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 ...
Child Thriving After Becoming World's Youngest to Receive Gene Therapy for AADC Deficiency
Sriansh Ojha was fully into his "terrible twos" on an April afternoon at Cincinnati Children's as he squealed and chased his ...
The American Journal of Managed Care1d
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 Reference1d
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 ...
FiercePharma1d
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...
Daily6h
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 ...