Association Française contre les Myopathies (AFM), Paris
25
4
5
15
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
Moderate Risk
Score: 35/100
0.0%
0 terminated/withdrawn out of 25 trials
100.0%
+13.4% vs industry average
16%
4 trials in Phase 3/4
0%
0 of 15 completed trials have results
Key Signals
Enrollment reporting
Analytics
Activity Timeline
Global Presence
Clinical Trials (25)
Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease
Role: collaborator
Standard of Care Comparative Arm of Phase 1/2 Gene Therapy Trial DREPAMIR" in Severe Sickle Cell Disease Patients
Role: collaborator
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Role: collaborator
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.
Role: collaborator
Polygraphy at Home for Neuromuscular Patients Under Non-invasive Mechanical Ventilation
Role: collaborator
Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
Role: collaborator
Wheelchair Positioning and Neuromuscular Diseases
Role: collaborator
Natural History Study of Children With LAMA2-related Dystrophies
Role: collaborator
Cell Signaling, Reinnervation and Metabolism in Kennedy Disease and Amyotrophic Lateral Sclerosis (ALS)
Role: collaborator
Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study
Role: collaborator
Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes
Role: collaborator
Impact of the Type of Interface in Neuromuscular Patients Treated With Nocturnal Noninvasive Ventilation
Role: collaborator
Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive
Role: collaborator
Clinical Validation Protocol for BCI for the Communication of Patients Suffering From Neuromuscular Disorders.
Role: collaborator
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
Role: collaborator
Prospective Study for 24-months of Physical Training Introduced in Lifestyle of Patients With FSHD : Tolerance, Sustainability and Efficiency of Unsupervised Training Program.
Role: collaborator
Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts
Role: collaborator
Study of Morphology and Functional Magnetic Resonance Imaging (MRI) Muscle Patients With Muscular Dystrophy Type FSHD Benefiting a Physical Training Introduced.
Role: collaborator
Study of Dopamine and Serotonin Transporters in Patients With Amyotrophic Lateral Sclerosis and Controls
Role: collaborator
Physical Training Introduction in Lifestyle of Facioscapulohumeral Dystrophy Patients
Role: collaborator