@parentprojectmd Avatar @parentprojectmd Parent Project Muscular Dystrophy

Parent Project Muscular Dystrophy posts on YouTube about community, in the, dyne therapeutics, hosted the most. They currently have [-----] followers and [---] posts still getting attention that total [---] engagements in the last [--] hours.

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Social Influence

Social category influence stocks 32% finance 4% travel destinations 2% social networks 1% technology brands 1% countries 1%

Social topic influence community 20%, in the 13%, dyne therapeutics #7, hosted 10%, study 7%, aoc 5%, gene 5%, university of 5%, breakout 5%, $ptct 5%

Top accounts mentioned or mentioned by @gmailcom

Top assets mentioned Dyne Therapeutics, Inc. (DYN) PTC Therapeutics, Inc. (PTCT) Wave Life Sciences Ltd. Ordinary Shares (WVE) Sarepta Therapeutics, Inc. (SRPT) PepGen Inc. (PEPG) Insmed, Inc. (INSM)

Top Social Posts

Top posts by engagements in the last [--] hours

"Webinar: Avidity Biosciences - EXPLORE44 Clinical Trial Investigating AOC [----] (Feb 2023) Avidity Biosciences joined PPMD for a community webinar on February [--] [----] to discuss the EXPLORE44 clinical trial. EXPLORE44 is an ongoing Phase 1/2 clinical trial evaluating AOC [----] for the treatment of individuals with Duchenne mutations amenable to exon [--] skipping. AOC [----] is an antibody oligonucleotide conjugate (AOC) and is designed to use an antibody to deliver an exon 44-skipping oligonucleotide to muscle and heart cells. AOC [----] is the first of multiple AOCs Avidity is developing for the"
YouTube Link 2023-02-13T21:03Z [----] followers, [----] engagements

"Webinar: Capricor Therapeutics HOPE-3 Community Update PPMD recently held a community webinar with Capricor Therapeutics during which the company shared their most recent results from the Phase [--] HOPE-3 trial evaluating deramiocel (CAP-1002) Capricors investigational cell therapy for the treatment of Duchenne muscular dystrophy. The presentation included a review of the data a discussion of planned next steps for FDA engagement and regulatory strategy and a dedicated Q&A period"
YouTube Link 2025-12-22T19:27Z [----] followers, [----] engagements

"Webinar: Avidity Biosciences - Topline Data from EXPLORE44 Clinical Trial Avidity Biosciences recently joined PPMD for a community webinar to share topline data from their EXPLORE44 clinical trial which is assessing the safety and efficacy of the investigational therapy delpacibart zotadirsen (formerly AOC [----] abbreviated as del-zota) in people living with Duchenne mutations amenable to exon [--] skipping. The Avidity team shared background information about del-zota and the EXPLORE44 study provided an overview of results and answered questions from the community. Disclaimer: This webinar"
YouTube Link 2025-04-01T19:56Z [----] followers, [---] engagements

"Webinar: Sarepta Therapeutics - Community Update on ELEVIDYS On January [--] [----] Sarepta Therapeutics joined PPMD for a breaking news community webinar to discuss the recently released three-year top line data for ELEVIDYS Sareptas gene therapy for the treatment of Duchenne. The presentation included a review of the data a timely discussion of what this means for the community and next steps as well as a dedicated Q&A period"
YouTube Link 2026-01-29T16:29Z [----] followers, [---] engagements

"Webinar: Dyne Therapeutics The Clinical Impact of the FORCE Platform for Duchenne Dyne Therapeutics joined PPMD on Wednesday June [--] [----] for a community webinar on the DELIVER clinical trial evaluating DYNE-251. The Dyne team provided the latest updates on the Phase 1/2 global DELIVER study of the companys investigational therapy for individuals living with Duchenne who are amenable to exon [--] skipping"
YouTube Link 2025-06-10T18:55Z [----] followers, [---] engagements

"Restoring Dystrophin via Exon Skipping Strategies -- PPMD [----] Annual Conference Restoring Dystrophin via Exon Skipping Strategies AOC [----] - Mark Stahl MD (Avidity Biosciences) BMN [---] - Dave Jacoby MD PhD (BioMarin Pharmaceutical Inc.) DYNE-251 - Ash Dugar PhD MBA (Dyne Therapeutics) ED051 - Michelle Mellion MD (PepGen Inc.) ENTR-601-44 - Nerissa Kreher MD MBA (Entrada Therapeutics) SRP-5051 - Teji Singh MD (Sarepta Therapeutics) WVE-N531 - Mike Panzara MD MPH (Wave Life Sciences)"
YouTube Link 2022-08-15T20:35Z [----] followers, [---] engagements

"PPMD [----] Annual Conference: Day [--] - Dystrophin: The Missing Muscle Protein Dystrophin: The Missing Muscle Protein Moderator: Eric Camino PhD Understanding Dystrophin Expression Stan Nelson MD Dystrophin Restoration Pipeline Discussing Avidity Biosciences Biomarin Pharmaceuticals Dyne Therapeutics Entrada Therapeutics NS Pharma Pepgen WAVE Life Sciences Kevin Flanigan MD From the Bench: Novel Approaches to Dystrophin Restoration John Lueck PhD Q&A"
YouTube Link 2025-07-08T14:59Z [----] followers, [---] engagements

"Webinar: RGX-202 - AFFINITY DUCHENNE Pivotal Program and Functional Data During the community webinar REGENXBIO provides an update on its AFFINITY DUCHENNE trial of RGX-202 a next-generation microdystrophin gene therapy. REGENXBIOs Dr. Naz Dastgir discusses the companys pivotal trial plans and shares interim clinical data including the first functional data for RGX-202"
YouTube Link 2024-11-21T16:25Z [----] followers, [---] engagements

"Webinar: Dyne Therapeutics Topline Results from Phase 1/2 DELIVER Trial Dyne Therapeutics joined PPMD for a community webinar on January 28th at 1:00 PM ET. The webinar features a presentation with Dr. Doug Kerr Dyne's Chief Medical Officer who shares topline results from the DELIVER clinical trial along with new long-term data showing sustained benefits out to [--] months. DELIVER is Dynes global randomized placebo-controlled double-blind Phase 1/2 trial that evaluated the safety tolerability and efficacy of zeleciment rostudirsen (z-rostudirsen also known as DYNE-251) in individuals with"
YouTube Link 2026-02-09T19:44Z [----] followers, [---] engagements

"Edgewise Therapeutics: Update on Sevasemten Program in Becker and Duchenne Muscular Dystrophies Edgewise Therapeutics joined PPMD for a community webinar on Friday June [--] [----] for an update on the companys sevasemten program in Duchenne and Becker. The Edgewise team reviewed new Open-label Extension MESA data in Becker as well as interim data from the ongoing LYNX and FOX Phase [--] studies"
YouTube Link 2025-07-02T19:05Z [----] followers, [---] engagements

"Webinar: Avidity Biosciences - EXPLORE44-OLE Topline and Functional Data Presentation Avidity Biosciences joined PPMD for a community webinar on November [--] [----] during which the company shared topline and functional data from their EXPLORE44-OLE clinical trial. Their investigational therapy delpacibart zotadirsen (abbreviated as del-zota) is in development for individuals with Duchenne muscular dystrophy who are amenable to exon [--] skipping"
YouTube Link 2025-11-26T14:43Z [----] followers, [---] engagements

"Sarepta Therapeutics: Safety Update on ELEVIDYS in Non-Ambulatory Individuals Living with Duchenne Sarepta Therapeutics recently joined PPMD for a community webinar to share details from the companys recent safety update on ELEVIDYS and discuss steps the company is proactively taking in response to the second reported case of acute liver failure in a non-ambulatory patient resulting in death following treatment with ELEVIDYS. Following the presentation Sarepta answered questions from the community"
YouTube Link 2025-06-19T19:17Z [----] followers, [----] engagements

"Breakout Session: Emerging Therapies for Becker Muscular Dystrophy - PPMD [----] Annual Conference EDG-5506 - Joanne Donovan MD (Edgewise Therapeutics) Vamorolone (ReveraGen) - Paula Clemens MD (University of Pittsburgh) Becker Imaging Study - Krista Vandenborne PT PhD (University of Florida) Becker Muscular Dystrophy Becker Muscular Dystrophy"
YouTube Link 2022-08-12T12:43Z [----] followers, [----] engagements

"The Superhighway: Investing in Duchenne - PPMD [----] Connect Conference "The Superhighway: Investing in Duchenne" presented by Sharon Hesterlee PhD at PPMD's 20th Annual Connect Conference. Download a PDF of Dr. Hesterlee's slides or an MP3 of his talk: http://www.parentprojectmd.org/site/PageServerpagename=Connect_conference_presentations_14"
YouTube Link 2014-09-30T20:12Z [----] followers, [---] engagements

"Webinar: Interim Results from FORWARD-53 Study of WVE-N531 in Duchenne Muscular Dystrophy (Oct 2024) PPMD hosted a community webinar with Wave Life Sciences on Wednesday October [--] [----]. The webinar featured Waves President and CEO and Chief Development Officer who presented an update on the recently announced interim results from the company's ongoing Phase [--] FORWARD-53 study of WVE-N531 in individuals with Duchenne who are amenable to exon [--] skipping"
YouTube Link 2024-10-03T18:54Z [----] followers, [---] engagements

"Webinar: NS Pharma Announcement of VILTEPSO (August 2020) NS Pharma joined PPMD for a webinar on August [--] [----] to provide an update about VILTEPSO from Ben Yungher PhD NS Pharmas Medical Science Liaison and its associated patient support program from Randy McGonigal NS Pharmas Director of Market Access"
YouTube Link 2020-09-03T18:01Z [----] followers, [----] engagements

"Parent Project Muscular Dystrophy: Duchenne PSA In the PSA called "Have You Seen This Boy" Ms. Dukakis helps visitors understand the initial symptoms of Duchenne "If you see a boy who struggles to walk or climb stairs who has enlarged calves who pulls himself up in a different way and can't seem to keep up with his peers you could be noticing symptoms of Duchenne muscular dystrophy.""
YouTube Link 2007-08-16T14:16Z [----] followers, 25K engagements

"Advisory Committee Meetings 101: A Conversation with Tim Franson MD Following the news of the May 12th FDA Advisory Committee meeting planned for SRP-9001 (delandistrogene moxeparvovec) Sareptas investigational gene therapy for Duchenne muscular dystrophy PPMD hosts an important discussion with FDA expert Tim Franson around what Advisory Committee Hearings are all about"
YouTube Link 2023-05-02T22:56Z [----] followers, [---] engagements

"PPMD's End Duchenne Tour: ESPAOL - Sarepta Presentaciones de patrocinadores - Sarepta. View more from PPMD's End Duchenne Tour: Spanish Remote Series at parentprojectmd.org/spanishtour"
YouTube Link 2019-09-27T15:32Z [----] followers, [---] engagements

"PPMD [----] Conference - Inflammation and Fibrosis [----] marks PPMDs 25thAnnual Conference. No other Duchenne conference comes close to the experience of the PPMD Annual Conference. Families physicians researchers caregivers industry partners and those living with Duchenne gather to discuss all topics Duchenne. They gather to connect to share information and to discuss and debate the latest opportunities in Duchenne research. This conference is like no other. Here the key is community.All pieces of this incredible community come together to learn to listen and to look forward at driving change."
YouTube Link 2019-07-12T17:38Z [----] followers, [---] engagements

"Webinar: Wave Life Sciences - Update on FORWARD-53 Study of WVE-N531 Wave Life Sciences recently joined PPMD for a community webinar to provide an update on the companys Phase [--] FORWARD-53 trial of WVE-N531 Waves exon skipping oligonucleotide being investigated for the treatment of individuals with Duchenne who are amenable to exon [--] skipping. Wave discussed the recently released FORWARD-53 data and answered questions from the community. Disclaimer: This webinar program is intended solely to provide information to the public. The views and opinions expressed as part of this webinar program"
YouTube Link 2025-04-08T13:57Z [----] followers, [---] engagements

"Webinar MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Duchenne - June [----] PPMD and Dr. Joanne Donovan hosted a webinar on June [--] [----] to review information on edasalonexent (previously known as CAT-1004) and the MoveDMD trial. The positive results from Part A of the MoveDMD trial and the design and inclusion criteria of Part B (Phase 2) of the trial that is currently running were reviewed. There was also an opportunity to ask questions"
YouTube Link 2016-06-11T15:01Z [----] followers, [----] engagements

"Webinar PTC Therapeutics Provides Regulatory Update on Translarna - August [----] On August [--] [----] PPMD hosted a webinar with PTC Therapeutics to discuss the latest news regarding Translarna. Craig McDonald presented the benefits of Translarna from emerging data and senior leadership from PTC were on hand to answer questions regarding the path forward for the therapy with the FDA. If you or your loved one have experience with Translarna we encourage you to fill out our Patient Experience Survey so we can continue to compile the experiences of our community with this therapy:"
YouTube Link 2016-08-05T13:13Z [----] followers, [---] engagements

"In the Pipeline: Pre-Clinical Gene Therapy (PPMD's [----] Virtual Annual Conference)"
YouTube Link 2021-07-22T16:15Z [----] followers, [---] engagements

"Trials in Duchenne - Sarepta PPMD's [----] Connect Conference Dana Martin Pharm.D from Sarepta Therapeutics presents at PPMD's [----] Connect Conference on June [--] 2016"
YouTube Link 2016-07-28T14:18Z [----] followers, [---] engagements

"Webinar: Overview of WVE-N531 and the FORWARD-53 Clinical Trial with Wave Life Sciences Feb [----] Wave Life Sciences joined PPMD for a community webinar on February [--] [----] for an update on the companys FORWARD-53 study. Wave provided an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular dystrophy who are amenable to exon [--] skipping as well as an overview of the potentially registrational FORWARD-53 study"
YouTube Link 2024-02-22T17:44Z [----] followers, [---] engagements

"Webinar SareptAssist Patient Services Overview - September [----] On September [--] [----] Parent Project Muscular Dystrophy and Sarepta Therapeutics Inc. hosted an informational webinar about SareptAssist Sarepta's patient support program. To learn more visit www.SareptAssist.com"
YouTube Link 2016-10-03T14:58Z [----] followers, [---] engagements

"Catabasis (CAT 1004) PPMD's [----] Connect Conference Speakers: Joanne Donovan MD PhD"
YouTube Link 2015-07-17T15:22Z [----] followers, [---] engagements

"Becker Breakout Session: Clinical Care -- PPMD [----] Annual Conference Clinical Care Moderator: Rachel Schrader MS APRN CPNP-PC Neuromuscular Care: Craig McDonald MD Cardiology: Jon Soslow MD MSCI Genetic Counseling: Niki Armstrong MS CGC Physical Therapy: Abby Dierker PT DPT"
YouTube Link 2023-07-19T15:08Z [----] followers, [---] engagements

"Trial Update: Edasalonexent - Catabasis PPMD's [----] Connect Conference Joanna Donovan MD PhD (Catabasis) presents at PPMD's Annual Connect Conference on June [--] 2017"
YouTube Link 2017-07-17T16:55Z [----] followers, [---] engagements

"In the Pipeline: Dystrophin Restoration (PPMD's [----] Virtual Annual Conference)"
YouTube Link 2021-07-22T16:31Z [----] followers, [---] engagements

"How You Can Participate in the Sarepta Ad Comm The Duchenne community is working closely together to prepare for the April [--] Advisory Committee Meeting for Sareptas therapy eteplirsen. PPMD the Duchenne Alliance the Center for DMD at UCLA Jett Foundation and the Race to Yes have put together a short video so that you can learn about the different ways you can become involved in this historic event. Whether submitting written testimony attending the Ad Comm in person participating in the Open Public Hearing or doing all three this community is strongest when we work together and collaborate."
YouTube Link 2016-03-15T12:57Z [----] followers, [---] engagements

"CAT-1004 (Catabasis) Clinical Trial Webinar May [----] On May [--] [----] Dr. Joanne Donovan Chief Medical Officer at Catabasis Pharmaceuticals presented information about MoveDMD: an upcoming Phase 1/2 clinical trial with CAT-1004 in boys with Duchenne muscular dystrophy. Catabasis May [----] Catabasis May 2015"
YouTube Link 2015-05-29T20:23Z [----] followers, [----] engagements

"Understanding CK Levels: An Introduction to Creatine Kinase (CK) Levels"
YouTube Link 2025-12-16T20:39Z [----] followers, [---] engagements

"Recently Reported Updates - PTC Therapeutics (PPMD's [----] Virtual Annual Conference)"
YouTube Link 2021-07-22T16:04Z [----] followers, [---] engagements

"Webinar: SRP-9001 Data Update from Studies [---] [---] and [---] (August 2022) PPMD invited Sarepta Therapeutics to present a SRP-9001 gene therapy data update on August [--] [----] following their recent presentation at a medical conference in July"
YouTube Link 2022-09-14T19:34Z [----] followers, [----] engagements

"PPMD [----] Conference - Restoring or Replacing Dystrophin The [----] PPMD Annual Conference marks the 24th time families physicians researchers caregivers industry partners and those living with Duchenne have gathered to discuss all topics Duchenne. This conference is like no other. Here the key is community. All the pieces of this incredible community come together to learn to listen and to look forward at driving change. Every single voice is important and every moment is rich. Now is the time for us to come together as a community and seize this moment. The field is ripe with opportunity and"
YouTube Link 2018-07-19T16:32Z [----] followers, [---] engagements

"Research Row Breakout Session: Impacting Downstream -- PPMD [----] Annual Conference Research Row: Impacting Downstream Capricor Therapeutics (Cap-1002) Edgewise Therapeutics (EDG-5506) FibroGen (Pamrevlumab) Antisense Therapeutics (ATL1102) Cumberland Pharmaceuticals (Ifetroban)"
YouTube Link 2023-07-19T15:09Z [----] followers, [---] engagements

"In the Pipeline: Gene Therapy - Sarepta Therapeutics Sarepta Therapeutics - Our Pipeline: Micro-dystrophin Gene Therapy"
YouTube Link 2020-09-02T18:01Z [----] followers, [----] engagements

"In the Pipeline: Gene Therapy - Pfizer Inc. Pfizer's investigational gene therapy (PF-06939926) for Duchenne muscular dystrophy"
YouTube Link 2020-09-02T18:00Z [----] followers, [----] engagements

"Webinar: ENVISION Study Update with Sarepta Therapeutics Sareptas Medical Director Clinical Development discusses the companys upcoming ENVISION study for SRP-9001"
YouTube Link 2023-06-16T16:50Z [----] followers, [----] engagements

"Summit Therapeutics (C1100) PPMD's [----] Connect Conference Speakers: Mike Boss PhD"
YouTube Link 2015-07-21T04:30Z [----] followers, [---] engagements

"Community Update Call Sarepta & Summit - October [----] Sarepta Therapeutics and Summit recently announced a license and collaboration agreement which grants Sarepta the right to commercialize Summit's utrophin modulators in Europe should they receive approval. This includes ezutromid which is currently in the Phase [--] PhaseOut DMD trial. PPMD hosted a community update call with Sarepta and Summit on October [--] [----]. Learn more about this partnership on our website: http://community.parentprojectmd.org/profiles/blogs/sarepta-therapeutics-and-summit-enter-into-exclusive-license-and- community"
YouTube Link 2016-10-18T14:35Z [----] followers, [---] engagements

"Trials in Duchenne - Pfizer PPMD's [----] Connect Conference Mike Binks MD PhD from Pfizer presents at PPMD's [----] Connect Conference on June [--] 2016"
YouTube Link 2016-07-28T14:18Z [----] followers, [---] engagements

"Webinar MoveDMD Trial: Catabasis Provides Update - February [----] The Catabasis team spoke to our community on February [--] [----] to discuss what they learned from this latest trial and what comes next in the MoveDMD program. catabasis edasalonexent movedmd clinical trial research treatment duchenne duchenne muscular dystrophy dmd dbmd catabasis edasalonexent movedmd clinical trial research treatment duchenne duchenne muscular dystrophy dmd dbmd"
YouTube Link 2017-02-06T16:45Z [----] followers, [---] engagements

"Recently Reported Updates - FibroGen (PPMD's [----] Virtual Annual Conference)"
YouTube Link 2021-07-22T16:06Z [----] followers, [---] engagements

"2018 PPMD Conference - Friday June 29th - Afternoon Session LIVE POLLING If you are a patient caregiver or family member please go to http://PollEv.com/PPMD to participate live. The [----] PPMD Annual Conference marks the 24th time families physicians researchers caregivers industry partners and those living with Duchenne have gathered to discuss all topics Duchenne. This conference is like no other. Here the key is community. All the pieces of this incredible community come together to learn to listen and to look forward at driving change. Every single voice is important and every moment is"
YouTube Link 2018-06-30T02:08Z [----] followers, [----] engagements

"Sarepta Therapeutics (Eteplirsen) PPMD's [----] Connect Conference Speakers: Ed Kaye MD Eteplirsen Eteplirsen"
YouTube Link 2015-07-17T15:15Z [----] followers, [----] engagements

"In the Pipeline: Restoring Dystrophin - Sarepta Therapeutics (RNA Targeted Therapies) Sarepta Therapeutics - RNA Targeted Therapies for Duchenne muscular dystrophy patients"
YouTube Link 2020-09-02T18:01Z [----] followers, [----] engagements

"Genetics Basics: The DMD Gene"
YouTube Link 2025-06-02T15:45Z [----] followers, [---] engagements

"Webinar PolarisDMD: Phase [--] Clinical Trial of Edasalonexent a Novel NF-kB Inhibitor - Nov [----] PPMD and Catabasis hosted a webinar on November [--] [----] to discuss the Phase [--] PolarisDMD clinical trial studying edasalonexent in Duchenne muscular dystrophy which is enrolling boys ages [--] to [--] (up to 8th birthday) regardless of mutation type who have not been on steroids for at least [--] months"
YouTube Link 2018-11-16T19:23Z [----] followers, [---] engagements

"Family Friday: When Youre a Carrier Learning that you are a female carrier of Duchenne can be shocking overwhelming and confusing. During PPMD's June [--] [----] Family Friday on Facebook Live we were joined by a panel of female carriers. We discussed the importance of caring for yourself both physically and mentally including the theme of resilience and what it means for caregivers of children and adults living with Duchenne. duchenne muscular dystrophy carrier female dmd duchenne muscular dystrophy carrier female dmd"
YouTube Link 2020-06-29T12:41Z [----] followers, [---] engagements

"Webinar: Viltepso An FDA-Approved Treatment for Patients Amenable to Exon [--] Skipping (March 2023) PPMD and NS Pharma hosted a community webinar on March [--] [----] to discuss VILTEPSO an FDA-approved treatment for patients with Duchenne amenable to exon [--] skipping. Mathula Thangarajh MD joined us to discuss the clinical evidence to support the early initiation of treatments in Duchenne and provide an overview of the role of exon skipping. Dr. Thangarajh also described the safety and tolerability profile of VILTEPSO and discussed the efficacy of VILTEPSO for the treatment of Duchenne. Note: This"
YouTube Link 2023-05-02T22:57Z [----] followers, [---] engagements

"Trials in Duchenne - Catabasis PPMD's [----] Connect Conference Joanne Donovan MD PhD from Catabasis presents at PPMD's [----] Connect Conference on June [--] 2016"
YouTube Link 2016-07-28T14:18Z [----] followers, [---] engagements

"Webinar: Dyne Therapeutics Update on DYNE-251 (July 2022) Dyne Therapeutics joined PPMD for a community webinar on Wednesday July [--] [----]. Dyne provided an update on the recent U.S. Food and Drug Administration (FDA) announcement lifting the DYNE-251 clinical hold and clearing the Investigational New Drug (IND) application. Dyne will initiate a clinical trial of DYNE-251 in individuals with Duchenne amenable to skipping exon [--] and expects to begin dosing trial participants in mid-2022"
YouTube Link 2022-08-22T14:43Z [----] followers, [---] engagements

"BioMarin (Drisapersen) PPMD's [----] Connect Confernece Speakers: Giles Campion MD PhD BioMarin Pharmaceutical (Business Operation) Health (Industry) BioMarin Pharmaceutical (Business Operation) Health (Industry)"
YouTube Link 2015-07-17T15:17Z [----] followers, [---] engagements

"Gene Therapy Town Hall -- PPMD [----] Annual Conference Gene Therapy Town Hall (AAV mediated approaches) Moderator: Tim Cripe MD PhD Buddy Cassidy - Immune Challenges - Barry Byrne MD PhD Gene Therapy and the Heart - Chet Villa MD Solid Biosciences REGENXBIO Pfizer Inc Sarepta Therapeutics Panel Discussion and Q & A"
YouTube Link 2023-07-19T15:09Z [----] followers, [---] engagements

"Webinar: Edgewise Therapeutics A Novel Approach Designed to Protect Muscle in Becker (May 2023) Edgewise Therapeutics joined PPMD for a community webinar on May [--] [----] to discuss the companys novel approach designed to protect muscle in individuals living with Becker muscular dystrophy. The webinar highlighted EDG-5506 an investigational compound in the form of a daily oral pill that could be used alone or in combination with other therapies regardless of mutation. Our presenters also discussed results from the ongoing open-label ARCH study in adults with Becker muscular dystrophy and"
YouTube Link 2023-05-11T22:35Z [----] followers, [----] engagements

"Webinar: Affinity Duchenne and Affinity Beyond: Updates on Investigational RGX-202 from REGENXBIO During this PPMD community webinar REGENXBIO provides updates on AFFINITY DUCHENNE their investigational gene therapy for Duchenne muscular dystrophy and AFFINITY BEYOND an AAV8 antibody assessment study"
YouTube Link 2023-10-13T20:04Z [----] followers, [---] engagements

"Webinar: Sarepta Therapeutics - Duchenne Gene Therapy Update Sarepta Therapeutics recently joined PPMD for a community webinar to share the latest updates on their gene therapy program. In addition to program updates Dr. Chet Villa cardiologist at Cincinnati Childrens Hospital and Dr. Craig McDonald of UC Davis an investigator in the EMBARK study of ELEVIDYS offered clinical perspectives"
YouTube Link 2025-04-28T19:25Z [----] followers, [----] engagements

"Webinar NS Pharma Provides Viltolarsen Update - November [----] On November [--] [----] NS Pharma joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of its investigational drug viltolarsen (NS-065/NCNP-01) including its mechanism of action and regulatory status. NS Pharma also provided an update on the viltolarsen clinical program including the completed Phase [--] Phase 1/2 and Phase [--] studies as well as an update on the viltolarsen Phase [--] trial (RACER53) including anticipated countries where study will be conducted study design description of the"
YouTube Link 2019-11-26T19:40Z [----] followers, [---] engagements

"Webinar: Edgewise Therapeutics - Clinical Trials in Becker Muscular Dystrophy Edgewise Therapeutics joined PPMD to share more about Edgewises ongoing clinical trials which aim to protect muscle in individuals living with Becker. This webinar features Edgewises Chief Scientific Officer and Chief Medical Officer who shares two year results from the completed Phase [--] ARCH study in adults with Becker and more information about their current enrolling trial GRAND CANYON a phase [--] pivotal cohort for adults living with Becker"
YouTube Link 2024-08-09T15:17Z [----] followers, [---] engagements

"Edgewise Therapeutics: A Novel Approach Designed to Protect Muscle in Becker Muscular Dystrophy Edgewise Therapeutics joined PPMD for a community webinar on October [--] [----] to discuss the companys investigational compound EDG-5506. Joanne M. Donovan M.D. Ph.D. Chief Medical Officer at Edgewise and Alan Russell Ph.D. Edgewises Co-Founder and Chief Scientific Officer provided an overview of results from the ongoing open-label ARCH study in adults with Becker muscular dystrophy and shared information about Edgewises currently enrolling trial GRAND CANYON a phase [--] trial for adults living with"
YouTube Link 2023-10-24T18:11Z [----] followers, [---] engagements

"Webinar: Advancing DYNE-251 - An Investigational Medicine for Duchenne to Deliver for Patients Dyne Therapeutics joined PPMD for a presentation of clinical data from the DELIVER Trial of DYNE-251 in individuals living with Duchenne muscular dystrophy who are amenable to exon [--] skipping first released on May 20th [----]. These data demonstrated compelling impact on key disease biomarkers and trends in improvement of multiple functional endpoints"
YouTube Link 2024-08-20T14:36Z [----] followers, [---] engagements

"Webinar: Pulmonary Management in Winter Season (Dec 2022) As RSV Flu and COVID-19 continue to be on the rise its critical we understand how to manage respiratory symptoms at home when to seek emergency medical care and how to advocate for yourself or your child if admitted to the hospital. On December [--] [----] PPMD hosted a community webinar featuring three expert pulmonologists to discuss respiratory management in Duchenne to help ensure you are as prepared as possible this winter season"
YouTube Link 2022-12-21T17:39Z [----] followers, [---] engagements

"Webinar: Overview of Topline Results from Phase [--] EMBARK Study PPMD was joined by Crystal Proud MD for a community webinar on November 8th to discuss the recently shared Phase [--] topline results of the EMBARK study (SRP-9001-301). Siobhan Fitzgerald Executive Director of Patient Affairs at Sarepta Therapeutics also joined the webinar to answer frequently asked questions related to plans moving forward"
YouTube Link 2023-11-13T21:16Z [----] followers, [----] engagements

"PPMD [----] Annual Conference: Day [--] - Research Row: Approaches to Alter Progression Research Row: Approaches to Alter Progression Leanne Ward MD FRCPC Childrens Hospital of Eastern Ontario (Moderator) Roche - Satralizumab: Maitea Guridi PhD Roche Capricor Therapeutics - CAP-1002: Mark Awadalla Capricor Therapeutics Edgewise Therapeutics - EDG-5506: Joanne Donovan MD PhD Edgewise Therapeutics Univ. of Florida - Tadalfil: Tanja Taivassalo PhD University of Florida Panel Discussion & Q&A"
YouTube Link 2024-07-24T18:34Z [----] followers, [---] engagements

"Becker Breakout Session: Emerging Therapies -- PPMD [----] Annual Conference Emerging Therapies in Becker Moderator: Eric Camino PhD Edgewise Therapeutics: Joanne Donovan MD University of Pittsburgh: Paula Clemens MD ImagingNMD Study: Claudia Senesac PT PhD PCS"
YouTube Link 2023-07-19T15:08Z [----] followers, [---] engagements

"Webinar Introducing PPMO Sarepta Therapeutics May [----] On May [--] [----] Sarepta Therapeutics Inc. joined Parent Project Muscular Dystrophy for a webinar update to discuss PPMO Sarepta's next generation precision RNA-targeting therapeutic platform for use in Duchenne muscular dystrophy. Sarepta also introduced their planned innovative enhancements to the trial design for their actively enrolling Phase [--] clinical trial 5051-101 for their PPMO exon [--] candidate (SRP-5051 NCT: 03375255) and outlined their plans for future development of additional exon targets. * This webinar recording contains"
YouTube Link 2018-05-31T19:26Z [----] followers, [----] engagements

"In the Pipeline: Gene Therapy - Solid Biosciences Solid Biosciences - Microdystrophin Gene Therapy (SGT-001)"
YouTube Link 2020-09-02T18:00Z [----] followers, [---] engagements

"Novel Approaches for Combatting Duchenne -- PPMD [----] Annual Conference Novel Approaches for Combatting Duchenne ASP0367 - Ha Tran MD MS FAAP (Astellas Pharma) CAP-1002 - Linda Marban PhD (Capricor Therapeutics) EDG-5506 - Joanne Donovan MD (Edgewise Therapeutics)"
YouTube Link 2022-08-15T20:34Z [----] followers, [---] engagements

"In the Pipeline: Downstream Targets - Astellas Pharma Astellas Pharma - ASP0367 Development Program Update for Duchenne Muscular Dystrophy"
YouTube Link 2020-09-02T18:01Z [----] followers, [---] engagements

"Research Row Breakout Session: Exon Skipping Strategies - PPMD [----] Annual Conference Research Row: Exon-Skipping Strategies Sarepta Therapeutics ( SRP-5051) Dyne Therapeutics (DYNE-251) PepGen (EDO51) BioMarin Pharmaceutical (BMN351) Wave Life Sciences (WVE-N531) Entrada Therapeutics (ENTR-601-44) Avidity Biosciences (AOC 1044) NS Pharma (NS-089)"
YouTube Link 2023-07-19T15:09Z [----] followers, [---] engagements

"PPMD [----] Annual Conference: Day [--] - Exploring the Gene Therapy Landscape Exploring the Landscape of Gene Therapy Moderator: Eric Camino PhD Gene Therapy Overview: How We Got Here and Where We Are Going Jeff Chamberlain PhD Understanding Immune Responses Craig McDonald MD Gene Therapy Pipeline: Discussing Insmed REGENXBIO Sardocor Solid Biosciences Kevin Flanigan MD Q&A"
YouTube Link 2025-07-08T15:02Z [----] followers, [---] engagements

"Webinar: Dyne Therapeutics Advancing FORCE in the Pursuit of Transformative Therapies (April 2023) Dyne Therapeutics joined PPMD for a community webinar on April [--] [----] to discuss Dyne's FORCE platform. Ashish Dugar PhD MBA Dyne's Chief Medical Affairs Officer and Molly White Dyne's Vice President Global Head of Patient Advocacy and KOL Engagement discussed Dynes DELIVER clinical trial a Phase 1/2 study evaluating DYNE-251 for the treatment of individuals with Duchenne muscular dystrophy who have mutations amenable to exon [--] skipping"
YouTube Link 2023-05-02T22:55Z [----] followers, [---] engagements

"Webinar: Transforming the Treatment Paradigm with Dyne Therapeutics (March [--] 2021) Dyne Therapeutics joined PPMD for a webinar on March [--] [----] to provide an overview of the company mission its novel FORCE platform designed to revolutionize drug delivery to muscle and the company's Duchenne muscular dystrophy drug development program"
YouTube Link 2021-04-07T16:47Z [----] followers, [---] engagements

"Webinar Community Discussion with Sarepta Therapeutics - August [----] On August [--] [----] PPMD invited Sarepta leaders to join a community discussion regarding the New Drug Application for golodirsen. We apologize if you were unable to access the webinar audio due to the high number of attendees. We look forward to learning more about next steps as Sarepta and the FDA work to bring our community a safe effective therapy. 8-21-19 8-21-19"
YouTube Link 2019-08-22T14:54Z [----] followers, [----] engagements

"In the Pipeline: Protecting the Muscle (PPMD's [----] Virtual Annual Conference)"
YouTube Link 2021-07-22T16:27Z [----] followers, [---] engagements

"PPMD [----] Annual Conference: Day [--] - Clinical Trials: A Panel Discussion on the Developmental Path Erica Goude MSCCRP UC Davis Medical Center (Moderator) Paolo Bettica MD PhD Italfarmaco Naz Dastgir DO RegenxBIO Joanne Donovan MD PhD Edgewise Therapeutics Kevin Flanigan MD Nationwide Children's Hospital Erik Henricson PhD MPH UC Davis Health"
YouTube Link 2024-07-24T18:34Z [----] followers, [---] engagements

"Webinar: Edgewise Therapeutics - A Novel Approach to Protect Muscle in Duchenne & Becker (Oct 2022) PPMD was joined by Edgewise Therapeutics for a community webinar on October [--] [----]. Edgewise's Chief Scientific Officer and Chief Medical Officer discussed the clinical development program for EDG-5506 a myosin modulator in Becker and Duchenne muscular dystrophies. The webinar also highlighted the mechanism of action results from the ongoing open-label ARCH study in adults with Becker muscular dystrophy and upcoming trials"
YouTube Link 2022-10-21T18:59Z [----] followers, [----] engagements

"PPMD [----] Annual Conference: Day [--] - Research Row: Strategies to Restore Dystrophin Research Row: Strategies to Restore Dystrophin Annemieke Aartsma-Rus PhD University of Leiden (Moderator) Avidity Biosciences - AOC 1044: Henry Nchienzia PhD Avidity Biosciences Biomarin - BMN 351: Peter Velzquez PhD BioMarin Pharmaceutical Inc. Dyne Therapeutics - DYNE-251: John Cox MBA Dyne Therapeutics Entrada Therapeutics - ENTR-601-44: Mahasweta Girgenrath PhD Entrada Therapeutics NS Pharma - NS-089 / NS-050: Robert Crozier PhD NS Pharma Pepgen - PGN-EDO51: Bassem Morcos MD Pepgen PTC Therapeutics -"
YouTube Link 2024-07-24T18:34Z [----] followers, [---] engagements

"Webinar Capricor Therapeutics' HOPE-Duchenne Clinical Trial - November [----] Capricor Therapeutics joined PPMD for a webinar on November [--] [----] to discuss the 12-month results from Capricor's HOPE-Duchenne Clinical Trial as well as the companys plans for the HOPE-2 Clinical Trial in the first quarter of [----] pending regulatory approval"
YouTube Link 2017-12-04T20:38Z [----] followers, [---] engagements

"Webinar: Twice Weekly Steroids & Exercise Study Update and Preliminary Findings (April 2022) On April [--] [----] Dr. Tanja Taivassalo joined PPMD for a community webinar to present an update on the University of Floridas Department of Defense funded clinical trial evaluating the potential of low-dose twice weekly steroids and exercise training as therapeutic strategies for individuals with Duchenne. Dr. Taivassalo shared preliminary findings and an update on the study which aims to determine whether use of the recommended daily dose of steroid taken only twice a week may be as effective with"
YouTube Link 2022-04-29T15:17Z [----] followers, [---] engagements

"Webinar: Dyne Therapeutics Advancing DYNE-251 an Investigational Medicine for DMD Jan [----] Dyne Therapeutics joined PPMD for a community webinar on January [--] [----]. to present initial clinical data released earlier this month from the DELIVER trial of DYNE-251 in individuals with Duchenne muscular dystrophy who are amenable to exon [--] skipping. Additionally Dyne discussed its proprietary FORCE platform driving efforts to develop targeted modern oligonucleotide therapeutics while aiming to overcome limitations in delivery to muscle tissue with the goal of stopping or reversing disease"
YouTube Link 2024-02-14T18:10Z [----] followers, [---] engagements

"SMT C1100 - Phase [--] trial results with Summit PMC (Dec 2012) Webinar Summit Webinar Summit"
YouTube Link 2012-12-13T20:20Z [----] followers, [---] engagements

"Webinar: A Conversation with PTC Therapeutics (Dec 2022) PTC Therapeutics joined PPMD for a community webinar on Friday December [--] [----] for a discussion regarding PTCs history with the community and plans for the future including activities to bring ataluren (Translarna) to US patients with nonsense mutations"
YouTube Link 2022-12-20T16:16Z [----] followers, [---] engagements

"Webinar NS Pharma's Exon [--] Skipping Program - November [----] On November [--] [----] Parent Project Muscular Dystrophy and NS Pharma (US subsidiary of Nippon Shinyaku Co.Ltd) hosted a webinar to discuss NS Pharma's Exon [--] Skipping Program. Dr. Clemens and Lauren Morgenroth reviewed data from the Phase I study conducted in Japan and presented the study design for the trial that is opening in the US later this month"
YouTube Link 2016-11-22T16:55Z [----] followers, [---] engagements

"Approved Therapies - Sarepta Therapeutics SareptAssist: Support for Duchenne Patients"
YouTube Link 2020-09-02T18:01Z [----] followers, [---] engagements

"Webinar Capricor's HOPE-2 Interim Analysis - July [----] On July [--] [----] PPMD and Capricor hosted a webinar to discuss updates on Capricor's HOPE-2 clinical program"
YouTube Link 2019-07-19T15:19Z [----] followers, [---] engagements

"Webinar: Sarepta Therapeutics - Data update from MOMENTUM study SRP-5051 Mar [----] On March [--] [----] Sarepta Therapeutics joined PPMD for a community webinar: Data Update From the MOMENTUM Study of SRP-5051. Sarepta Therapeutics was invited to share data from Part B of MOMENTUM a Phase [--] Study of SRP-5051 in people living with Duchenne muscular dystrophy who are amenable to skipping exon 51"
YouTube Link 2024-03-19T15:31Z [----] followers, [---] engagements

"In the Pipeline: Next-Gen Exon Skipping (PPMD's [----] Virtual Annual Conference)"
YouTube Link 2021-07-22T16:15Z [----] followers, [----] engagements

"Webinar: Viltepso - A Treatment Option for Patients Amenable to Exon [--] Skipping (April 2021) Parent Project Muscular Dystrophy was joined by NS Pharma for a webinar on April [--] [----] to learn more about VILTEPSO a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to exon [--] skipping and its associated patient support program"
YouTube Link 2021-05-03T18:30Z [----] followers, [----] engagements

"Webinar Vamorolone Clinical Trials in Duchenne - January [----] On January [--] [----] ReveraGen joined Parent Project Muscular Dystrophy for a webinar to provide the community with an overview of the vamorolone drug development program for Duchenne including results showing improved muscle function in completed clinical trials in [--] Duchenne boys and a description of an ongoing clinical trial of [---] Duchenne boys that is accepting new patients at [--] sites internationally. 1-30-19 1-30-19"
YouTube Link 2019-02-12T19:17Z [----] followers, [----] engagements

"Gene Therapy Approaches to Replace or Restore Dystrophin -- PPMD [----] Annual Conference Gene Therapy Approaches to Replace or Restore Dystrophin PF-06939926 - Kasia Lobello MD (Pfizer Inc.) RGX-202 - Jahannaz Dastgir DO (REGENXBIO) scAAV9.U7-ACCA - Kevin Flanigan MD (Nationwide Children's Hospital) SGT-001 - Carl Morris PhD (Solid Biosciences) SRP-9001 - Teji Singh MD (Sarepta Therapeutics)"
YouTube Link 2022-08-15T20:12Z [----] followers, [---] engagements

"PPMD [----] Annual Conference: Day [--] - Approved Therapies & Sequential Therapy Approved Drugs and Sequential Therapy: Discussing Catalyst Pharmaceuticals ITF Therapeutics NS Pharma PTC Therapeutics Sarepta Therapeutics Moderator: Eric Camino PhD Leigh Ramos-Platt MD Aravindhan Veerapandiyan MD Larry Markham MD FAAP FACC Sam Mackenzie MD PhD Craig McDonald MD"
YouTube Link 2025-07-08T15:02Z [----] followers, [---] engagements

"PPMD [----] Annual Conference: Day [--] - Nurturing Mental Wellness in Everyday Life Nurturing Mental Wellness in Everyday Life Kristy Lewandowski MA CCC-SLP"
YouTube Link 2025-07-08T15:02Z [----] followers, [--] engagements

"Genetic Variants and Treatment Pathways"
YouTube Link 2025-06-02T15:46Z [----] followers, [---] engagements

"PPMD [----] Annual Conference: Day [--] - Trials & Treatments: Making Decisions that are Right for You Making Decisions That Are Right For You (Panel Discussion) Moderator: Sue Apkon MD Panelists: Maru Chapur Winston Choi Chris Bennett Eli Perez Jonah Perez"
YouTube Link 2025-07-08T15:02Z [----] followers, [---] engagements

"Understanding CK Levels: Interpreting Creatine Kinase Levels in the Clinic"
YouTube Link 2025-11-21T16:33Z [----] followers, [---] engagements

"PPMD [----] Annual Conference: Day [--] - Therapies that Slow Progression Therapies that Slow Progression Moderator: Eric Camino PhD Understanding Cell Therapies: What They Are What They Arent April Pyle PhD Tackling Other Issues with Novel Therapies Discussing Cumberland Pharmaceuticals Capricor Therapeutics Edgewise Therapeutics and Roche Larry Markham MD FAAP FACC Aravindhan Veerapandiyan MD Q&A"
YouTube Link 2025-07-08T14:59Z [----] followers, [---] engagements

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