MDS-PAS y EHDN: Actualización sobre la enfermedad de Huntington, desde la epidemiología hasta el manejo y el asesoramiento Course Format Start Date End Date Regional Online Course Fri, Jul 7, 2023 Fri, Jul 28, 2023 Program Description El curso MDS-PAS y EHDN: Actualización sobre la enfermedad de Huntington, desde la epidemiología hasta el manejo […]
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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 […]
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 […]
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 […]
extract1_04/2023_NL-issue48
Extract from the newsletter: Issue 48 ”Rocking It Up: An Interview with Flaviano Giorgini” […]
March 23rd: HUNTINGTON’s Gratitude Day 2023
On March 23rd, 1993, scientists announced the discovery of the gene causing Huntington’s Disease (HD). […]
EHDN Newsletter – 48th 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 […]
HDYO International Young Adults Congress
March 17-19, 2023 in Glasgow, Scotland […]
CHDI’s 18th Annual Huntington’s Disease Therapeutics Conference
… to be held April 24 through 27, 2023, in Dubrovnik, Croatia. The conference program will cover all aspects of HD drug development, including the latest advances in understanding HD pathophysiology, novel HD therapeutic approaches, current and emerging biomarkers, and updates on ongoing clinical trials. […]
28 February, 2023 IS RARE DISEASE DAY
Raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and carers. […]