Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 35/100

Failure Rate

0.0%

0 terminated/withdrawn out of 23 trials

Success Rate

100.0%

+13.5% vs industry average

Late-Stage Pipeline

17%

4 trials in Phase 3/4

Results Transparency

0%

0 of 15 completed trials have results

Key Signals

6 recruiting

Enrollment Performance

Analytics

N/A
6(42.9%)
Phase 3
4(28.6%)
Phase 2
3(21.4%)
Phase 1
1(7.1%)
14Total
N/A(6)
Phase 3(4)
Phase 2(3)
Phase 1(1)

Activity Timeline

Global Presence

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Clinical Trials (23)

Showing 20 of 23 trials
NCT07432867Phase 1Recruiting

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

NCT05785637Recruiting

Polygraphy at Home for Neuromuscular Patients Under Non-invasive Mechanical Ventilation

Role: collaborator

NCT01648634Phase 3Completed

Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

Role: collaborator

NCT07049588Not ApplicableRecruiting

Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease

Role: collaborator

NCT05006859Recruiting

Wheelchair Positioning and Neuromuscular Diseases

Role: collaborator

NCT06354790Recruiting

Natural History Study of Children With LAMA2-related Dystrophies

Role: collaborator

NCT05107349Not ApplicableRecruiting

Cell Signaling, Reinnervation and Metabolism in Kennedy Disease and Amyotrophic Lateral Sclerosis (ALS)

Role: collaborator

NCT02780492Completed

Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study

Role: collaborator

NCT05408702Completed

Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes

Role: collaborator

NCT03458507Not ApplicableCompleted

Impact of the Type of Interface in Neuromuscular Patients Treated With Nocturnal Noninvasive Ventilation

Role: collaborator

NCT01225614Phase 3Unknown

Efficacy and Tolerance of Early Launching of Nocturnal Non Invasive

Role: collaborator

NCT02284022Completed

Clinical Validation Protocol for BCI for the Communication of Patients Suffering From Neuromuscular Disorders.

Role: collaborator

NCT01302600Phase 2Completed

Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.

Role: collaborator

NCT01689480Completed

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

NCT00773227Phase 2Completed

Treatment of Dysphagia in Oculopharyngeal Muscular Dystrophy by Autologous Transplantation of Myoblasts

Role: collaborator

NCT01990976Completed

Study of Morphology and Functional Magnetic Resonance Imaging (MRI) Muscle Patients With Muscular Dystrophy Type FSHD Benefiting a Physical Training Introduced.

Role: collaborator

NCT01160263Phase 3Completed

Study of Dopamine and Serotonin Transporters in Patients With Amyotrophic Lateral Sclerosis and Controls

Role: collaborator

NCT02141724Completed

Wheelchair Positioning and Pain in Neuromuscular Patients

Role: collaborator

NCT01116570Not ApplicableCompleted

Physical Training Introduction in Lifestyle of Facioscapulohumeral Dystrophy Patients

Role: collaborator

NCT00847847Not ApplicableCompleted

Neuromuscular Transmission in Amyotrophic Lateral Sclerosis

Role: collaborator