Clinical trials: Congenital Myasthenic Syndrome
Lambert Eaton Myasthenic Syndrome (LEMS)
Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP
Lambert Eaton Myasthenic Syndrome (LEMS) is rare neurological disorder that results in muscle weakness and limited reflex activity.
- Trial name
- NCT01378546
- Testing
- 3,4-diaminopyridine
- Where
- United States
- Sponsor
- Louis H. Weimer, MD
Congenital Myasthenic Syndrome
Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes
The study tests the notion that patients suffering from certain types of congenital myasthenic syndromes are benefitted by the use of Albuterol at doses used in clinical practice.
- Trial name
- NCT01203592
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Mayo Clinic
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenia (CM)
3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)
Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disorder which affects the nerve-muscle junction. The major symptoms of LEMS are progressive muscle weakness.
- Trial name
- NCT02012933
- Testing
- 3,4-diaminopyridine
- Where
- United States
- Sponsor
- Oregon Health and Science University
Myasthenic Syndromes, Congenital
Ephedrine for the Treatment of Congenital Myasthenia
Previous research has demonstrated possible efficacy of Ephedrine in the treatment of congenital myasthenia caused by end-plate acetylcholinesterase deficiency.
- Trial name
- NCT00541216
- Testing
- Ephedrine
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Hadassah Medical Organization
Congenital Myasthenic Syndrome
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7…
- Trial name
- NCT06078553
- Where
- 33 sites in United States, Austria and 10 more
- Volunteers
- 152 people
- Sponsor
- argenx