uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline Read more…

HD Community Insights: Knowledge, Empathy, and Encouragement – The EHA Conference Inspired Me

Almost exactly 18 months after my diagnosis of suffering from Huntington’s Disease, I embarked on the journey to the European Huntington Conference in Belgium. The days in Blankenberge became an informative and occasionally overwhelming experience for me. Community, compassion, and empowerment – these terms roughly describe my memories of those Read more…

Empowering Communities: A Recap of Recent Initiatives by the Brazilian Huntington Association (ABH)

The Brazilian Huntington Association (ABH – Associação Brasil Huntington) is incredibly active and has been conducting numerous initiatives for the community. This level of commitment is vital because it not only benefits those affected by the disease but also raises the much-needed awareness about Huntington’s Disease. This article serves as Read more…

PTC Therapeutics Shares Positive Interim Data from PIVOT-HD Clinical Trial in Huntington’s Disease Patients

June 21, 2023 PDF Version – Dose-dependent lowering of blood Huntingtin (HTT) protein levels at 12 weeks –– Favorable tolerability profile with no treatment-related serious adverse events or NfL spikes –  – Conference call and webcast to be held June 21st at 8:00 am EDT –  SOUTH PLAINFIELD, N.J., June 21, 2023 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared Read more…

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