Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.
Benitec Biopharma will present interim clinical study results for BB-301 gene therapy in oculopharyngeal muscular dystrophy patients at the Muscular Dystrophy Association Conference on March 9, 2026, including 12-month and 24-month follow-up data.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07505342 |
Remote Assessments and Genetic Determinants of Myotonic Dystrophy |
RECRUITING | |
| NCT06165172 |
Early Feasibility Study of the MyoRegulator® for Treatment of ALS |
COMPLETED | NA |
| NCT05989620 |
Long-Term Development of Muscular Dystrophy Outcome Assessments |
RECRUITING | |
| NCT05600764 |
The Natural History of TRPV4 Neuropathy |
RECRUITING | |
| NCT05412394 |
Once Weekly Infant Corticosteroid Trial for DMD |
RECRUITING | PHASE4 |
| NCT04193085 |
Wearable Technology to Assess Gait Function in SMA and DMD |
COMPLETED | |
| NCT03777319 |
Spironolactone Versus Prednisolone in DMD |
TERMINATED | PHASE1 |
| NCT03659422 |
Dietary Approach to Improving Quality of Life in Amyotrophic Lateral Sclerosis |
WITHDRAWN | NA |
| NCT03655223 |
Early Check: Expanded Screening in Newborns |
ENROLLING_BY_INVITATION | |
| NCT03639701 |
Treatment of TK2 Deficiency With Thymidine and Deoxycytidine |
ACTIVE_NOT_RECRUITING | PHASE1/PHASE2 |
