Wave Life Sciences Announces Positive Update from Phase 1b/2a SELECT-HD Trial with Initial Results Indicating Allele-Selective Target Engagement with WVE-003 in Huntington’s Disease

September 20, 2022 at 7:30 AM EDT PDF Version Single doses of WVE-003 appear generally safe and well-tolerated CSF mutant huntingtin (mHTT) protein was reduced following single doses of 30 or 60 mg; mean mHTT reduction across both cohorts was 22% (median reduction 30%) from baseline 85 days post-single dose قراءة المزيد……

uniQure Announces Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT (mHTT) observed in cerebral spinal fluid (CSF) at 12 months in evaluable patients treated with AMT-130 ~ ~ Neurofilament Light قراءة المزيد……

Annexon Biosciences updates trial results

ANNEXON BIOSCIENCES REPORTS PHASE 2 CLINICAL TRIAL RESULTS DEMONSTRATING UPSTREAM CLASSICAL COMPLEMENT INHIBITION ASSOCIATED WITH CLINICAL BENEFIT IN HUNTINGTON’S DISEASE On June 7, Annexon, that is a clinical-stage biopharmaceutical company developing a new class of medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced promising, final قراءة المزيد……

Webinar – Explore for Huntington: The Adventure of Dimitri Poffé in Latin America

To finish May Awareness Month in the most inspiring and hopeful way possible, the International Huntington Association and European Huntington Association had a webinar with Dimitri Poffé, a Huntington’s Disease advocate that is moving the community while traveling South America all by bike. Dimitri is Huntington positive but he hasn’t قراءة المزيد……

Poland and Belgium spearhead efforts to help Ukrainian HD families 

If the situation in Ukraine isn’t desperate enough, spare a thought for those with the added burden of Huntington’s Disease. Danuta Lis, president of the Polish HD Association and Bea De Schepper, vice president of the Belgian Huntington Liga (Flanders) have been leading efforts to help them.  As fighting broke out قراءة المزيد……

أخبار عاجلة – خطط روش لعمل دراسات جديدة على دواء تومينيرسين

Some encouraging news has just come in from Roche.  Analysis of the results of Roche’s Phase III GENERATION HD1 trial, which was stopped in March 2021, has found that a subset of patients may benefit from Tominersen.  The analysis showed that less frequent dosing of Tominersen may benefit younger patients قراءة المزيد……

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