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 Leer más…

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 Leer más…

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 Leer más…

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