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 ...
emjreviews.com17h
FDA approves first gene therapy delivered directly to the brain
The drug is a treatment for a rare and fatal genetic disorder called aromatic l-amino acid decarboxylase (AADC) deficiency, ...
FiercePharma17h
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 ...
FDA Grants Conditional Approval For PTC Therapeutics' Gene Therapy As First Ever With Direct Administration In Brain
PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...
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 ...
Pharmabiz4h
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., ...
Monthly Prescribing Reference12h
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 ...
The Pharma Letter18h
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 ...
ClearPoint Neuro Announces FDA De Novo Marketing Authorization of SmartFlow Cannula for Direct Delivery of Gene Therapy to the Brain
Modifying Treatment for AADC Deficiency in the United States SOLANA BEACH, CA / ACCESSWIRE / November 13, 2024 / ...
BioSpace19h
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 ...
ClearPoint Neuro announces SmartFlow Neuro Cannula FDA authorization
ClearPoint Neuro (CLPT) “announced the U.S. Food and Drug Administration has granted marketing authorization for the SmartFlow Neuro ...