Key Highlights

Risk & Performance

Pipeline Risk Assessment

Pipeline Risk Assessment

Based on historical performance

Moderate Risk

Score: 45/100

Failure Rate

5.1%

2 terminated/withdrawn out of 39 trials

Success Rate

92.6%

+6.1% vs industry average

Late-Stage Pipeline

5%

2 trials in Phase 3/4

Results Transparency

20%

5 of 25 completed trials have results

Key Signals

8 recruiting5 with results

Enrollment Performance

Analytics

Phase 2
7(36.8%)
Phase 1
6(31.6%)
N/A
3(15.8%)
Phase 4
2(10.5%)
Early Phase 1
1(5.3%)
19Total
Phase 2(7)
Phase 1(6)
N/A(3)
Phase 4(2)
+1 more

Activity Timeline

Global Presence

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Clinical Trials (39)

Showing 20 of 39 trials
NCT03655223Active Not Recruiting

Early Check: Expanded Screening in Newborns

Role: collaborator

NCT03489278Recruiting

Clinical Procedures to Support Research in ALS

Role: collaborator

NCT03458832Active Not Recruiting

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Role: collaborator

NCT07505342Recruiting

Remote Assessments and Genetic Determinants of Myotonic Dystrophy

Role: collaborator

NCT00272883Recruiting

Molecular and Genetic Studies of Congenital Myopathies

Role: collaborator

NCT03639701Phase 1Active Not Recruiting

Treatment of TK2 Deficiency With Thymidine and Deoxycytidine

Role: collaborator

NCT05989620Recruiting

Long-Term Development of Muscular Dystrophy Outcome Assessments

Role: collaborator

NCT05600764Recruiting

The Natural History of TRPV4 Neuropathy

Role: collaborator

NCT01203085Completed

Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

Role: collaborator

NCT01193088Recruiting

Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

Role: collaborator

NCT01193075Recruiting

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

Role: collaborator

NCT05412394Phase 4Recruiting

Once Weekly Infant Corticosteroid Trial for DMD

Role: collaborator

NCT06165172Not ApplicableCompleted

Early Feasibility Study of the MyoRegulator® for Treatment of ALS

Role: collaborator

NCT04193085Completed

Wearable Technology to Assess Gait Function in SMA and DMD

Role: collaborator

NCT03777319Phase 1Terminated

Spironolactone Versus Prednisolone in DMD

Role: collaborator

NCT01938495Phase 2Completed

Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS)

Role: collaborator

NCT01783509Completed

Limb Girdle Muscular Dystrophy (LGMD) Natural History

Role: collaborator

NCT02895789Completed

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

Role: collaborator

NCT00577577Phase 2Completed

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Role: collaborator

NCT00790582Phase 2Completed

A Multi-Center Controlled Screening Trial of Safety and Efficacy of Lithium Carbonate in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Role: collaborator