Neuromuscular Disorders

Patient cohort

NCT04668716 – Brain Involvement in Dystrophinopathies Part 2

NCT04583917 – Brain Involvement in Dystrophinopathies Part 1

NCT04281485 – Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT05558813 – Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP)

NCT04833894 – Evaluating the Pharmacokinetics Pharmacodynamics and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

C9orf72 zebrafish model

other zebrafish models

PBMCs from ALS patients (C9orf72 mutation)

Phenotypic characterization and drug screening platform for zebrafish (live imaging – in vivo electrophysiology recordings – and drug screening on zebrafish models)

TDP43 zebrafish model

PBMCs from SMA patients (SMN1 or SMN2 mutations)

Phenotypic characterization and drug screening platform for zebrafish (live imaging – in vivo electrophysiology recordings – and drug screening on zebrafish models)

NCT04833348 – Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies

NCT04668716 – Brain Involvement in Dystrophinopathies Part 2

NCT04583917 – Brain Involvement in Dystrophinopathies Part 1

NCT04283578 – Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome

NCT05032326 – Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial