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…

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

~ AMT-130 continues to be generally well-tolerated across both dose cohorts ~ ~ Patients treated with AMT-130 show preserved function compared to baseline and clinical benefits relative to natural history of the disease ~ ~ Neurofilament Light Chain (NfL) in cerebrospinal fluid (CSF) was below baseline at 24 months in Read more…

PRILENIA ANNOUNCE RESULTS FROM THE PROOF-HD STUDY: PROMISING, BUT MIXED RESULTS!

Today, April 25th, Prilenia announced results from the PROOF-HD clinical trial for Huntington’s Disease. Some of the participants showed significant benefits from the Pridopidine drug, while others did not benefit at all.  This is promising because for the first time we have a drug that demonstrates effect on disease progression, cognitive and motor Read more…

Prilenia Achieves Last Patient Last Visit in Phase 3 PROOF-HD Clinical Study for Huntington’s Disease

PROOF-HD is the only late-stage study in Huntington’s disease targeting clinical progression; topline results expected in early Q2 2023 NAARDEN, Netherlands and WALTHAM, Mass., 28 March 2023 – Prilenia Therapeutics B.V., a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases Read more…

Huntington’s Gratitude Day 2023

Dear friend,On March 23rd, 1993, scientists announced the discovery of the gene that causes Huntington’s Disease (HD). The announcement, after decades of research, made evident the partnership of ‘families living with HD’ and the ‘scientists working on their behalf’. This milestone and partnership were celebrated at homes, laboratories, and clinics.Around Read more…

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