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 vector-based gene therapy indicated for the treatment of adult and
FDA grants accelerated approval for PTC’s AADC deficiency gene therapy
This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency. The FDA's accelerated approval is based on the saf
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 a surgical procedure, and replaces the human dopa decarboxylase (DDC) gene that is mutated in the disease, allowing dopamine synthesis to take place.
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.,
FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency
The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.
PTC Therapeutics Gains Buy Rating Following FDA Approval of Groundbreaking Gene Therapy Kebilidi
William Blair analyst Sami Corwin has maintained their bullish stance on PTCT stock, giving a Buy rating on November 7. Sami Corwin has given
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 / ...
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 ...
pharmaphorum1y
PTC’s gene therapy for rare disease cleared for NHS use
NICE has recommended that PTC Therapeutics’ gene therapy for ultra-rare disease aromatic L-amino acid decarboxylase (AADC) deficiency Upstaza be made available for NHS use in draft guidance.