A Second Look: Checking in on uniQure's AMT-130 and PTC Therapeutics' PTC518 in Huntington's Disease

Executed on:Jan 24, 2025 9:15 AM EDT

Call Duration:30 minutes

Expert Profile

ExpertBlinded Expert

Institution: UCLA

Clinical Professor of Neurology. Director of Ataxia Clinic and Neurogenetics Clinical Trials at UCLA Medical Center.
Over 30 years of experience diagnosing and treating Friedreich's Ataxia;  has served on the National Ataxia Foundation's (NAF) Medical Research Advisory Board, NAF’s Board of Directors and is NAF’s Medical Director.
Helped develop the National Ataxia Database for clinical research and has participated in 2 natural history studies (for dominant Ataxia and Friedreich’s Ataxia) in addition to numerous clinical drug trials.

Interview Goal

This interview aims to explore the recent advancements and clinical data presented by uniQure and PTC Therapeutics regarding their AMT-130 and PTC518 programs for Huntington's Disease (HD). The focus will be on understanding the efficacy, safety, and potential impact of these treatments in slowing disease progression and improving patient outcomes.

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