Defunct Company
Total Trials
4
As Lead Sponsor
2
As Collaborator
Total Enrollment
40
NCT02453152
Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)
Phase: N/A
Role: Collaborator
Start: Oct 31, 2015
Completion: Jul 31, 2019
NCT03078881
Clinical Assessment Study in Crigler-Najjar Syndrome
Role: Lead Sponsor
Start: Mar 10, 2017
Completion: Jan 23, 2019
NCT03223194
Gene Transfer Clinical Study in Crigler-Najjar Syndrome
Phase: Phase 1
Start: Sep 8, 2017
Completion: Feb 11, 2021
NCT04240314
AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Phase: Phase 1/2
Start: Jan 15, 2020
Completion: Jul 1, 2025