Also known as: FA
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
Rocket Pharmaceuticals will participate in two investor conferences in March 2026, with the CEO taking part in a fireside chat and management hosting investor meetings at both events.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07649031 |
MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients |
RECRUITING | |
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Prenatal Transplantation for Fetuses With Fanconi Anemia |
NOT_YET_RECRUITING | PHASE1/PHASE2 |
| NCT07005297 |
Clinical Genetics Branch Eligibility Screening Survey |
NOT_YET_RECRUITING | |
| NCT06744283 |
Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia |
RECRUITING | |
| NCT05973656 |
Role of Acetaldehyde in the Development of Oral Cancer |
RECRUITING | NA |
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Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia |
RECRUITING | |
| NCT03351868 |
FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector |
RECRUITING | NA |
| NCT03050268 |
Familial Investigations of Childhood Cancer Predisposition |
RECRUITING | |
| NCT02720679 |
Investigation of the Genetics of Hematologic Diseases |
RECRUITING | |
| NCT00027274 |
Cancer in Inherited Bone Marrow Failure Syndromes |
RECRUITING |
