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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