GTX-102
- Generic Name
- GTX-102
- Brand Names
- -
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- -
- Unique Ingredient Identifier
- -
- Background
GTX-102 is an antisense oligonucleotide designed to target and inhibit the expression of UBE3A-AS. It is being investigated for the treatment of Angelman Syndrome.
- Associated Conditions
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- Associated Therapies
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RARE's Bone Disorder Drug Gets FDA's Breakthrough Therapy Tag
FDA grants Ultragenyx's setrusumab Breakthrough Therapy designation for osteogenesis imperfecta (OI) Type I, III, or IV, targeting fracture risk reduction in patients aged two and older. Setrusumab, a sclerostin inhibitor, is being evaluated in late-stage studies for OI, with preliminary data showing significant fracture rate reduction.
Acasti Announces Completion of Patient Enrollment in Pivotal Phase 3 STRIVE-ON Safety
Acasti Pharma completes patient enrollment in Phase 3 STRIVE-ON trial for GTX-104, an injectable nimodipine for aSAH, with data readout expected early 2025 and NDA submission planned for 1H 2025.
Related Clinical Trials:
Acasti Announces Completion of Patient Enrollment in
Acasti Pharma Inc. completed patient enrollment in its Phase 3 STRIVE-ON safety trial for GTX-104, an injectable nimodipine for aneurysmal subarachnoid hemorrhage (aSAH). The trial, involving 100 patients across 25 U.S. hospitals, compares GTX-104 with oral nimodipine. Acasti anticipates trial data in early 2025 and plans to submit a New Drug Application to the FDA in the first half of 2025.
Related Clinical Trials:
Drug Development Updates
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