Daily1h
FDA Approves First Gene Therapy for Treatment of AADC deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus ...
Pharmabiz11h
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., ...
The Pharma Letter1d
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 / / November 13, 2024 / ClearPoint Neuro, Inc. (Nasdaq:CLPT) (the 'Company'), a global therapy-enabling platform company ...
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 ...
Reuters1d
US FDA approves PTC Therapeutics' gene therapy for ultra-rare disorder
The therapy, sold under the brand name Kebilidi, is a one-time treatment for children and adults with an ultra rare inherited ...
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 ...
PTC Therapeutics, Inc.: PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
First-ever FDA approval for gene therapy directly administered to the brain - - Priority review voucher granted - - Broad label including children and adults - - ...
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 ...
Le Lézard1d
GiangisKitchen.com Shares Mouth-Watering Recipes that Can be Easily Prepared Days Before Thanksgiving
Popular recipe site GiangisKitchen.com has published a unique selection of recipes for delicious Thanksgiving appetizers, ...
BioWorld1d
PRV sweetens PTC approval of Kebilidi in AADC deficiency
PTC Therapeutics Inc.’s accelerated go-ahead from the U.S. FDA for Kebilidi (eladocagene exuparvovec-tneq), a recombinant adeno-associated virus serotype 2-based gene therapy to treat aromatic L-amino ...