HDAwareness2024

. #ICYMI doesnt this almost cure huntingtons disease? #askingforafriend #HDAwareness #CureHD #Huntingtonsdisease

Hoy tras nuestro productivo encuentro en el @CentroCREER, la comisión de incidencia política ha empezado a dar sus primeros pasos, escuchando, compartiendo ideas y definiendo objetivos en el corto y medio plazo para que el #Huntington empiece a estar en la agenda política. 💪🏻

Lynk Pharmaceuticals Achieves Regulatory Milestone: Zemprocitinib Advances in China for Atopic Dermatitis Treatment bit.ly/48jqt4P Read now at PatientWorthy.com #PatientWorthy #LynkPharmaceuticals #AtopicDermatitis #Zemprocitinib

Profiling the secretome: maternal obesity impacts redox and adipogenic signaling during neonatal mesenchymal stem cell adipogenesis doi.org/10.1093/stmcls…

It took a while, but eventually, columnist Tanita Allen learned that boundary-setting isn't about building walls; it's about gaining clarity. huntingtonsdiseasenews.com/columns/true-b… #HuntingtonsDiseaseNews #HuntingtonsDisease #RareDiseaseNews #Bionews

昨年立てた『月１本は映画館で映画を観る』と言う目標 会員になった #宮崎キネマ館 さんだけで２０本観ることが出来た 他の映画館併せたら約１年で計２４本 これまでと同じペースとは行かないかも知れませんが、続けて行こうと思います ＃終わりの鳥 と言う作品は円盤を買うぐらい良い出会いだった

あのイルカが20年ぶりに復活しました 当時は一部の質問に決まった回答しかできず、ひっそりと姿を消しました... 今回は対話型の生成AIを使用。性格を維持したまま賢く柔軟性を持っているとのことです news.web.nhk/newsweb/na/na-…

#Help4HDHIPE2026 is this weekend! We look forward to meeting up with our #HDFamily in #BaltimoreMD for a day filled with #HD #education ! Can't attend in person? Listen as we stream on Facebook LIVE to hear stories, insights, updates, & more!

From my friend @geneveritas "As the reportedly dysfunctional @US_FDA faces demands for greater clarity in the wake of rare-disease drug denials, uniQure continues to seek a path to get AMT-130, its historically efficacious Huntington’s disease gene therapy, approved. After reneging on its promise to allow uniQure to apply for AMT-130 approval in 2026, the FDA recommended a new, Phase III clinical trial. @SenRonJohnson, a Republican, has launched an investigation of the FDA, and a rare disease coalition has urged President Trump to restore regulatory clarity at the agency. A possible avenue to AMT-130 approval opened on April 14. Dr. Teresa Buracchio of the FDA said at the National Organization for Rare Disorders (NORD) symposium that the agency’s “plausible mechanism framework” for approval of bespoke gene therapies might be applied “to approve other therapies.” A bespoke therapy is given to a single individual, such as “Baby KJ,” the world’s first individual to be treated, and successfully, with a personalized (customized) CRISPR gene editing therapy. Dr. Buracchio stated that the FDA is open to applying the framework “conceptually” to Huntington’s disease broadly. A therapy needs to show “substantial evidence of effectiveness and a substantial improvement that’s clear and distinct from the natural history of the disease,” she said, noting that KJ demonstrated marked improvement in symptoms and reached developmental milestones. By comparison, a slowly progressive neurodegenerative disease like HD “is going to be a harder case to make,” Dr. Buracchio said. At the NORD symposium, uniQure’s Dr. David Margolin addressed the FDA’s concerns. Dr. Margolin stated that HD’s slow progression makes it nearly impossible to show clear efficacy over a short period. AMT-130 demonstrated a 75 percent slowing of HD progression over three years. Regarding the use of Enroll-HD patient registry data in place of a placebo in uniQure’s analyses, Dr. Margolin pointed out that the difference between AMT-130 clinical trial participants and the individuals uniQure selected from Enroll-HD was negligible. “I know there’s active dialogue with FDA and the Huntington’s disease organizations regarding how to interpret and best utilize these clinical scored measures, and that’s an ongoing process,” Dr. Margolin stated. In the current political and business climate, perhaps the HD community also needs a celebrity connection, as biotech observers have noted with dark humor. “I guess people living and dying with Huntington’s disease need an influencer/podcaster to text Trump,” said STAT senior biotech writer @adamfeuerstein, who shared an item about podcaster Joe Rogan receiving a promise from Trump of immediate FDA approval for a psychedelic treatment. “That’s how the FDA works these days.” Read more in my latest article." curehd.blogspot.com/2026/04/as-fda… @SenRickScott @SenateAging @FDACBER @houmanhemmati

@LogicalValueMx @Prof_Dollar @US_FDA @DrMakaryFDA @FDACDERDirector @houmanhemmati @SenCortezMasto @CortezMasto Thank you so much for looking into this matter, I hope the rest of the reps of our state will join you in getting some movement on this and AMT-130 @dinatitus @repdinatitus @MarkAmodeiNV2 @RepSusieLee @RepHorsford @SenJackyRosen

🎧 New conversation: ABC Executive Director Katie Sale speaks with @curedravet CEO Mary Anne Meskis and Patient Advocacy Director Shannon Cloud about the origins, advocacy work, and impact of their foundation. 👉 Listen sites.libsyn.com/420986/site/ep…

@US_FDA One important point I should have made in the video is that the surrogate marker, heparan sulfate, was reduced in the cerebrospinal fluid. A second important point is the treatment is given via a weekly IV infusion.

.. @LogicalValueMx $qure Tabrizi explains how they use real world data to compare AMT-130 to external control group (EnrollHD) and why regulators approve of if when done correctly.

Novartis' INVEST-HD Phase 3 trial: Site Initiations in USA and Canada buff.ly/0xKbqN0

@LogicalValueMx @US_FDA @DrMakaryFDA @FDACDERDirector @houmanhemmati . who is this Mr Kennedy? speaking in the post? Please and Thank You. 👍

$repl $qure From WSJ tonight @US_FDA @DrMakaryFDA @FDACDERDirector @houmanhemmati

HDSA provides support for everyone impacted by Huntington’s disease, including individuals living with HD, caregivers, and people at risk. For more information, visit: hdsa.org/.../community.… #LetsTalkAboutHD #HuntingtonsDisease #HDFamily #FamilyIsEverything

Stay Connected with HDSA on our social media pages! To continue receiving important updates, notifications, and community news from HDSA, please take a moment to sign up at: : app.donorview.com/Mqy33 #LetsTalkAboutHD #HDSAFamily #HDSA

HDSA is proud to announce the 2026 Request for Proposals for the HD Human Biology Project, and to introduce an exciting new companion program: the HD Human Experience Project. Learn More & Apply Below: hdsa.org/hd-research/hd… #LetsTalkAboutHD #Huntingtonsdisease #HDResearch

⏳💙 5 DAYS AWAY! 💙⏳ We are just 5 days away from the 2026 Huntington’s Disease Walk & Bike-a-thon in Dillon, SC! 👉 Register or donate here: give.hdsa.org/event/2026-bik… #hdawareness #CureHD #letstalkabouthd #huntingtonsdiseaseawareness #HDSA #EndHD #TogetherACure

Roche reports strong sales growth of +6% at constant exchange rates in the first quarter of 2026; -5% in CHF due to the significant appreciation of the Swiss franc. For more details, click here ➡️ spkl.io/6011AfHJR $ROG $RHHBY #FinancialResults