Kebilidi, a Gene Therapy for AADC Deficiency, Gets Accelerated Approval. Diana Ernst, RPh | Publish Date November 14, 2024 . Kebilidi is a one-time, recombinant adeno-associated virus- ...

FDA grants accelerated approval to PTC Therapeutics' Kebilidi, the first brain-administered gene therapy in the U.S. for AADC deficiency. Clinical trials show Kebilidi improved motor function in 8 ...

Kebilidi is the first FDA-approved gene therapy for the deficiency. PTC shares gained around 8.5 percent in the extended trading on the Nasdaq on Thursday, at $45.97.

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 ...

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 ...

UC San Diego scientists have created a gene therapy that goes beyond masking Alzheimer’s symptoms—it may actually restore ...

PTC Therapeutics’ Kebilidi™ (US) / Upstaza™ (ex-US) (eladocagene exuparvovec), an AAV vector-based gene therapy indicated for adults and children with aromatic12 L-amino acid decarboxylase ...