Category: News

ADORE-DH – Potential Stem Cell Therapy

ADORE-DH was a Phase II trial of a potential stem cell therapy for HD. Sponsored by AZIDUS Brasil (a full-service clinical research organization), this randomized, double-blind, placebo-controlled study aimed to […]

uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease

September 24, 2025 PDF Version ~ Pivotal study met primary endpoint; high-dose AMT-130 demonstrated statistically significant 75% disease slowing at 36 months as measured by cUHDRS compared to a propensity […]

Skyhawk Reports Data on Cohort C

Earlier today, Skyhawk provided the below update on Part C of their SKY-0515 Phase 1 trial. —-Community Letter—— Dear Skyhawk friends and colleagues, We are delighted to today announce the […]

Prilenia and Ferrer Provide Update on European Regulatory Process for Pridopidine in Huntington’s Disease

July 25, 2025 NAARDEN, Netherlands & WALTHAM, Mass. & BARCELONA, Spain–(BUSINESS WIRE)– Prilenia Therapeutics B.V. and Ferrer today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for […]

Huntington Academy, a new e-learning platform dedicated to HD, is now available

The European Huntington Association, with the support of a grant from the European Union, and in collaboration with the Ligue Huntington Francophone Belge, the Bulgarian Huntington Association and the ACHE […]

PTC518 PIVOT-HD Study Achieves Primary Endpoint

May 5, 2025 PDF Version – Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 – – Favorable dose-dependent trends across clinical scales in Stage 2 […]

May Awareness Month 2025 – Shining a light on HD: One Action a Day

Every May, communities around the world come together to shine a light on Huntington’s Disease (HD). From families and caregivers to clinicians, researchers, and advocates, we are united by one […]

UniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington’s Disease

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics License Application […]

Update on the Phase II GENERATION HD2 study

Roche has announced the continuation of the GENERATION HD2 Phase II clinical trial evaluating tominersen in individuals with early signs of Huntington’s disease. Following an interim analysis by an independent committee, no […]

A Look Back at the HDYO Congress 2025

The HDYO Congress 2025 brought together young people, families, researchers, and professionals from around the world for an unforgettable event dedicated to the Huntington’s Disease community. Over three inspiring days, […]