Congenital Myasthenic Syndrome

• CLINICAL_TRIALS research · 2026-04-23
  Clinical Trial Update: A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) [Phase 1]

  A Phase 1b study to investigate the safety and tolerability of ARGX-119 in adults with DOK7-congenital myasthenic syndrome (CMS) is currentl…

• CLINICAL_TRIALS research · 2026-03-13
  Clinical Trial Update: A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4

  A natural history study in participants with congenital myasthenic syndromes caused by mutations in DOK7, MUSK, AGRN, or LRP4 is currently r…

• CLINICAL_TRIALS research · 2025-11-10
  Clinical Trial Update: Patients With Congenital Myasthenic Syndrome Will be Treated With Mesenchymal Stem Cell Exosome Solution [Phase 1]

  Phase 1 clinical trial exploring mesenchymal stem cell exosome solution for treatment of congenital myasthenic syndrome; currently recruitin…

• CLINICAL_TRIALS approval · 2025-03-26
  Clinical Trial Update: Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

  Amifampridine phosphate has received marketing approval for Lambert-Eaton Myasthenic Syndrome and Congenital Myasthenic Syndrome. This updat…

• CLINICAL_TRIALS trial_result · 2016-01-01
  Clinical Trial Update: Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes [Phase 1]

  Phase 1 clinical trial update assessing the efficacy of albuterol for treating congenital myasthenic syndromes.

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• CLINICAL_TRIALS trial_result · 2011-11-18
  Clinical Trial Update: Pregnancy Outcomes in Congenital Myasthenie Syndrome

  This entry provides an update on the pregnancy outcomes from a completed clinical trial studying congenital myasthenic syndrome.