Medpage Today on MSN1d
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
The American Journal of Managed Care21h
FDA Approves Gene Therapy to Treat AADC Deficiency
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
Monthly Prescribing Reference16h
Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval
The Food and Drug Administration (FDA) has granted accelerated approval to Kebilidi™ (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid ...
FiercePharma22h
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 Dive15h
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 ...
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 ...
The Pharma Letter22h
FDA nod for AADC deficiency gene therapy from PTC
The US Food and Drug Administration (FDA) has granted accelerated approval of PTC Therapeutics’ gene therapy for the ...
Pharmabiz9h
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval
PTC Therapeutics’ gene therapy, Kebilidi to treat AADC deficiency receives US FDA approval: Warren, New Jersey Friday, November 15, 2024, 10:00 Hrs [IST] PTC Therapeutics, Inc., ...
pharmaphorum1d
PTC gets FDA okay for first brain-delivered gene therapy
Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during ...
PTC Therapeutics’ AADC deficiency gene therapy granted FDA approval
PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever ...
BioSpace1d
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 ...
Business Insider20h
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
The recent FDA approval of Kebilidi, a groundbreaking gene therapy for AADC deficiency, is a significant milestone for the company. It is the first gene therapy directly administered to the brain and ...