The FDA granted accelerated approval to eladocagene exuparvovec (Kebilidi) to treat children and adults with aromatic L-amino acid decarboxylase (AADC)

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First Brain-Delivered Gene Therapy Approved for AADC Deficiency

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The PTC Therapeutics gene therapy, Kebilidi, treats an enzyme deficiency that affects the body’s ability to produce dopamine, a neurotransmitter important for motor control. FDA approval for the product comes two years after it won its first regulatory approvals in Europe.  The post PTC Therapeutics Wins First FDA Approval for a Gene Therapy Dosed Directly Into the Brain appeared first on MedCity News.

MedCity News

PTC Therapeutics Wins First FDA Approval for a Gene Therapy Dosed Directly Into the Brain

PTC Therapeutics’ gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency deficiency has received FDA approval. It is the first-ever gene therapy approved in the U.S. that is directly administered to the brain. The treatment will be marketed under the name KEBILIDI (eladocagene exuparvovec-tneq). It is indicated for children and adults with AADC deficiency. AADC deficiency is an ultra-rare genetic disorder that interrupts the synt…

Inside Precision Medicine

PTC Nets AADC Gene Therapy Approval: First Brain-Delivered Gene Therapy in U.S.

The FDA has approved the first gene therapy for aromatic L-amino acid decarboxylase deficiency, a rare enzyme deficiency that leads to muscle and developmental issues. Developed by PTC Therapeutics, the gene therapy, known as eladocagene exuparvovec, is administered directly into the brain to replace the mutated DDC gene with a functioning version. The therapy has shown efficacy in pediatric patients, improving motor function and milestones in m…

Dataemia

PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US

Pharmaphorum

PTC gets FDA okay for first brain-delivered gene therapy

Learn about PTC Therapeutics' treatment for a rare and fatal genetic disorder called aromatic l-amino acid decarboxylase (AADC) deficiency

First Brain-Delivered Gene Therapy Approved for AADC Deficiency

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