US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
The U.S. Food and Drug Administration approved PTC Therapeutics' gene therapy to treat a potential fatal enzyme deficiency ...
FDA Approves PTC's Kebilidi As First Gene Therapy To Treat AADC Deficiency
The U.S. Food and Drug Administration approved PTC Therapeutics, Inc.'s Kebilidi (eladocagene exuparvovec-tneq) gene therapy for the ...
FiercePharma2d
PTC wins FDA approval for first brain-delivered gene therapy Kebilidi
The FDA issued its first stamp of approval for a cell or gene therapy back in 2017 to Novartis' Kymriah. | The gene therapy ...
BioPharma Dive1d
PTC wins US approval of gene therapy for fatal enzyme disorder
Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review ...
Logistics Management15d
Federal Railroad Administration issues Positive Train Control NPRM
The United States Department of Transportation’s Federal Railroad Administration (FRA) said yesterday it has published a notice of proposed rulemaking (NPRM) focused on regulations related to Positive ...
BioSpace2d
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene ...