PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...

The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...

PTC Therapeutics (PTCT) stock on watch as company receives FDA approval for gene therapy Kebilidi for AADC deficiency. Read ...

Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare ...

PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever ...

Enrollment and dosing complete in single ascending dose clinical trial of anti-tau antibody VY7523 for Alzheimer's; on track to generate top-line data H1 2025 - - Recent third-party clinical data esta ...

Elraglusib is undergoing investigation in an open-label, multicenter, phase 1/2 trial for the treatment of patients with ...

Actuate Therapeutics said its treatment for Ewing sarcoma was granted rare pediatric-disease designation from the Food and Drug Administration. Chief Executive Daniel Schmitt said early clinical data ...

Rare Pediatric Disease Designation is granted by the FDA for serious ... that could be utilized by the Company or potentially sold to another company for its use. About Actuate Therapeutics, Inc.

Relief Therapeutics announces positive final results of RLF-TD011 trial in epidermolysis bullosa: Geneva Tuesday, November 12, 2024, 10:00 Hrs [IST] Relief Therapeutics Holding SA ...

Oak Hill Bio, Chiesi announce first European patient enrolled in phase 2b study of OHB-607 for prevention of BPD in premature infants: Parma, Italy Tuesday, November 5, 2024, 14:0 ...