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10 results for “DOK7”

Trials

A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

100 participants · First posted Oct 12, 2023

Active, not recruiting

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

15 participants · First posted May 31, 2024

Not yet recruiting

A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)

105 participants · First posted Aug 4, 2026

Drugs

Investigational

AMP-101

Diakine-DK7 10 (EGFR)

News

argenx Advances ARGX-119 to Registrational Study for Ultra-Rare Congenital Myasthenic Syndromes

NIH Funding Cuts Under Trump Administration Threaten Early-Stage Biopharma Innovation

Andelyn Biosciences Partners with Amplo Biotechnology to Advance AAV Gene Therapies for Neuromuscular Junction Disorders