Category: News

An Update from LoQus23

LoQus23 Therapeutics is a biotechnology company investigating small molecule drugs that could stop the pathogenic triplet expansion that is the cause and driver of Huntington’s Disease (HD), myotonic dystrophy type […]

New Phase 1 POINT-HD Study

New Phase 1 POINT-HD study starts for selective huntingtin-lowering approach Dear Huntington’s community leaders, Following your request to receive updates about our research efforts, we are pleased to share that […]

Summary of “EHDN Platform Meeting on uniQure topline results: Unpacking the outcomes so far”

Warsaw, November 19th, 2025. Dear members, As you might know, yesterday the European Huntington’s Disease Network (EHDN) developed a seminar about the topline results of uniQure, a pharmaceutical company that […]

uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease

November 3, 2025 Dear Huntington’s Disease Community, We issued a press release today announcing that uniQure received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics […]

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 […]