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EHDN Newsletter – 49th edition

The EHDN newsletter (archive) aims to communicate the network’s activities and other developments of interest in the field of Huntington’s disease to the lay community, healthcare professionals and scientists. It appears three times a year (March 1, July 1, Nov 1). To receive EHDN news going forward, please subscribe to the EHDN news at the […]

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Good news from PTC and uniQure!

On 21 June 2023, positive findings from both PTC Therapeutics and uniQure were shared with the HD community on their respective huntingtin (HTT)-lowering approaches to treating HD. PTC’s interim 12-week data from the global PIVOT-HD trial of PTC518 showed dose-dependent lowering of HTT protein levels, confirmed the intended exposure in cerebrospinal fluid, and indicated a […]

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CHDI’s 18th Annual HD Therapeutics Conference – scientific presentations

CHDI’s 18th Annual HD Therapeutics Conference took place April 24 – 27, 2023, in Dubrovnik, Croatia, to allow more Europe-based colleagues to attend. This unique conference series focuses on drug discovery and development for Huntington’s disease, drawing participants from the biotech and pharmaceutical sectors as well as academia. The conference is intended as a forum […]

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UniQure Biopharma B.V. HD GeneTRX2 study endorsed* by EHDN

Study aims and goal: A Phase Ib/II Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington’s Disease. No. of study participants and sites: This phase Ib/II b study aims to recruit 15 participants from approximately 5 study […]

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Wave Life Sciences Ltd. SELECT-HD study endorsed* by EHDN

Study aims and goal: A Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP)-SNP3. No. of study participants and sites: This phase Ia/II b study aims to recruit 36 participants from approximately 25 […]

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Preliminary results of the Proof-HD trial

Prilenia has announced preliminary results of its PROOF-HD trial on pridopidine on Huntington’s disease. While the study missed both its primary endpoint (an improvement in the Unified Huntington Disease Rating Scale-Total Functional Capacity) and a key secondary endpoint by (Composite Unified Huntington’s Disease Rating Scale (cUHDRS)), the company was encouraged by positive signals in a […]

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