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@mike98572986
"@adgrx5 Nah just was posting the story if it is clearpoint related I will always put the symbol"
X Link @mike98572986 2025-10-15T17:05Z XXX followers, XX engagements
"$qure $clpt Victor Sung He notes that following the recent uniQure news his clinic has received more than XX calls a day from people wanting to learn more. Hes excited about whats next after this landmark study"
X Link @mike98572986 2025-10-14T23:23Z XXX followers, 7065 engagements
"$clpt Clearpoint isnt part of the study but shows advantages of ICV delivery and is a brand new study regarding ICV delivery comparison To our knowledge a comprehensive comparison study of these two routes of delivery in large animals has not been done for ASOs. Therefore we conducted a study to compare the ICV and IT-L route of delivery for the MALAT1 ASO in dogs. We delivered the ASO and assessed biodistribution and RNA knockdown (KD) across CNS regions. We found that ICV delivery resulted in substantial bilateral distribution of the ASO and KD. Further we found that ICV delivery resulted"
X Link @mike98572986 2025-10-13T17:17Z XXX followers, 1122 engagements
"$rgnx $clpt Neurodegenerative Disease: Clemidsogene lanparvovec (RGX-121) is a potential first-in-class treatment for MPS II also known as Hunter syndrome being developed and potentially commercialized in partnership with Nippon Shinyaku. In May 2025 the U.S. Food and Drug Administration (FDA) granted priority review of the BLA seeking accelerated approval for clemidsogene lanparvovec for MPS II; Prescription Drug User Fee Act (PDUFA) target action date of November X 2025. The FDA BLA review is progressing as planned; mid-cycle meeting and PLI and BIMO inspections were successfully completed."
X Link @mike98572986 2025-08-07T11:16Z XXX followers, 1343 engagements
"Impressive stuff Ive worked with these families for decades says Leone a professor of neuroscience and director of Rowan Universitys Cell and Gene Therapy Center. The results were seeing are impressive. Weve seen brain structures grow brain function improve; were seeing patients walk with minimal assistance. Their quality of life has completely changed. This is not the end but the beginning of a new era in the treatment of neurological disorders conditions that at their core involve impaired white matter she says noting that her ongoing research is motivated by the families affected by"
X Link @mike98572986 2025-10-15T15:18Z XXX followers, XXX engagements
"$qure $clpt Huntingtons Win Leaves More Patients Willing to Test The conversations have changed in the last X weeks with the prospect of a treatment people are more inclined to want to know whether they are at risk of getting sick says Miedzybrodzka CEO Matt Kapusta says the company hasnt made any firm decisions about what to do outside the US yet. Article is about people in North Scotland since they have a very high Huntingtons rate compared to other parts of the world. Most hospitals in the U.K. lack the kind of specialized equipment for the procedure according to Miedzybrodzka"
X Link @mike98572986 2025-10-10T16:55Z XXX followers, 1645 engagements
"$qure $clpt No wonder Sung was going so crazy last time he spoke I always thought he had only X patients under his care. He was seeing it in real time and couldnt say a word. Will be interesting to hear what he says this Sunday now that he can speak. Seven of the XX high-dose participants were treated at UAB where neurologist Dr. Victor Sung director of the Division of Movement Disorders and the UAB Huntingtons Disease Clinic called the results a huge step toward changing the course of a disease long considered untreatable. There recent procedures are only taking in the 8-10 hour range"
X Link @mike98572986 2025-10-10T23:52Z XXX followers, 3342 engagements
"$clpt $qure Awesome article on what the news means to the Huntingtons community "It is like winning the lottery XX times over" says Gemma XX. "I had accepted I would never get to grow old with my husband and that he might never see the kids become adults. That has all changed now." "I must have cried for three hours. Then the kids got home from school and I showed my daughter the BBC story and she burst into tears." "In some ways we are lucky he got past XX with no signs of it" says Gemma. "His mother died from the disease when she was XX. And now we have the prospect of a treatment. We have"
X Link @mike98572986 2025-09-27T17:56Z XXX followers, 2507 engagements
"$clpt $rgnx RGX-121 "I am highly encouraged by the 12-month pivotal data and continued safety and efficacy profile of RGX-121" said Roberto Giugliani M.D. Ph.D. ProfessorDepartment of Genetics UFRGS Medical Genetics Service HCPAPorto Alegre Brazil."The vast majority of Hunter syndrome patients have no current treatment options to address the neurodevelopmental decline of this disease and are in urgent need of new therapies and a one-time treatment option like RGX-121 could make a meaningful impact on their lives.""
X Link @mike98572986 2025-09-05T15:00Z XXX followers, 2038 engagements
"$qure $clpt Huntingtons Facebook group XXX votes XX percent yes on the question If a treatment for Huntingtons disease required brain surgery would you be willing to undergo it"
X Link @mike98572986 2025-10-05T03:51Z XXX followers, 4192 engagements
"Adds today $Dcth and $par Really liking par tech down here"
X Link @mike98572986 2025-09-30T16:19Z XXX followers, 1733 engagements
"$clpt $qure New presentation from Sarah Tabrizi minute XXXXX talks about uniqure procedure does say it is very complex they are getting it down to X hours and working on it. Working on shorter protocols. For children they can do it in 3-5 hours. Hour XX seconds talks about exon1 and why uniqure amt-130 is doing so well. @DesertDweller93 @Prof_Dollar @peter_mantas @PythiaR @biggercapital"
X Link @mike98572986 2025-10-03T16:38Z XXX followers, 10.1K engagements