Congenital myasthenic syndrome
MONDO:0018940Congenital myasthenic syndrome (CMS) is a group of genetic disorders of impaired neuromuscular transmission at the motor endplate characterized by fatigable muscle weakness.
Also known as: CMS, Congenital Myasthenic Syndromes, myasthenic syndrome, congenital, congenital MG, congenital myasthenia, erb-Goldflam syndrome, familial limb-girdle myasthenia, myasthenia gravis congenital
6 clinical trials for this condition and its sub-types, 6 tagged with Congenital myasthenic syndrome itself.
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Sub-types of Congenital myasthenic syndrome
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Postsynaptic congenital myasthenic syndrome 0 trials · 3 incl. sub-types
14 sub-types
- Congenital myasthenic syndrome 10 3 trials
- Congenital myasthenic syndrome 17 1 trial
- Congenital myasthenic syndrome 8 1 trial
- Congenital myasthenic syndrome 9 1 trial
- Congenital myasthenic syndrome 11 0 trials
- Congenital myasthenic syndrome 16 0 trials
- Congenital myasthenic syndrome 19 0 trials
- Congenital myasthenic syndrome 1A 0 trials Sub-types →
- Congenital myasthenic syndrome 2A 0 trials
- Congenital myasthenic syndrome 2C 0 trials
- Congenital myasthenic syndrome 3A 0 trials
- Congenital myasthenic syndrome 3B 0 trials
- Congenital myasthenic syndrome 3C 0 trials
- Congenital myasthenic syndrome 4 0 trials Sub-types →
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Presynaptic congenital myasthenic syndrome 0 trials · 1 incl. sub-types
10 sub-types
- Congenital myasthenic syndrome 8 1 trial
- Congenital myasthenic syndrome 18 0 trials
- Congenital myasthenic syndrome 19 0 trials
- Congenital myasthenic syndrome 20 0 trials
- Congenital myasthenic syndrome 21 0 trials
- Congenital myasthenic syndrome 6 0 trials
- Congenital myasthenic syndrome 7 0 trials
- Myasthenic syndrome, congenital, 23, presynaptic 0 trials
- Myasthenic syndrome, congenital, 24, presynaptic 0 trials
- Myasthenic syndrome, congenital, 25, presynaptic 0 trials
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Congenital myasthenic syndrome 15 0 trials
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Congenital myasthenic syndrome 5 0 trials
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3 sub-types
- Congenital myasthenic syndrome 12 0 trials
- Congenital myasthenic syndrome 13 0 trials
- Congenital myasthenic syndrome 14 0 trials
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Myasthenic syndrome, congenital, 22 0 trials
Most studied deeper sub-types
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One shot of gene therapy aims to fix a rare Muscle-Weakening disease
Cure Not yet recruitingResearchers are testing AMP-101, an experimental gene therapy, in people with DOK7 congenital myasthenic syndrome, a rare inherited disorder that causes muscle weakness and exercise intolerance. About 4 participants with moderate to severe DOK7 CMS will receive a single intraveno…
Phase 1 • Sponsor: Amplo Biotechnology • Aim: Cure
Last updated Sep 21, 2026 18:00 UTC
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Can a new infusion ease muscle weakness in rare genetic disorders?
Disease control Not yet recruitingThis Phase 3 trial is testing whether an experimental drug called adimanebart can help people aged 12 and older with certain congenital myasthenic syndromes (CMS) feel better and do daily activities more easily. Participants are randomly assigned to receive either adimanebart or …
Phase 3 • Sponsor: argenx • Aim: Disease control
Last updated Aug 06, 2026 00:00 UTC