Category: Research

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 […]

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 […]

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 […]

SOM Biotech announces Phase 2b study results

SOM Biotech presents the Phase 2b study results with SOM3355 demonstrating a unique profile with robust improvements of chorea in Huntington’s Disease Patients and a safe profile with no somnolence, […]

PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington’s Disease Program

December 2, 2024 – PTC to receive $1.0B in cash at closing –– PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones –– PTC to share profits in the U.S. and […]