The drug is a treatment for a rare and fatal genetic disorder called aromatic l-amino acid decarboxylase (AADC) deficiency, ...

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., ...

PTC Therapeutics' Kebilidi gains FDA accelerated approval as the first brain-administered gene therapy in the U.S. for AADC ...

This therapy is designed to address the needs of children and adults with aromatic L-amino acid decarboxylase (AADC) ...

The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...

most offering only symptomatic relief for a limited time by correcting the dopamine deficiency caused by loss of dopaminergic neurons, rather than targeting the neurodegenerative disease process ...

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 ...

PTC Therapeutics (PTCT) announced that the FDA accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever ...

Kebilidi is the first gene therapy approved by the FDA for administration directly into the brain, given via a cannula during ...

The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...

In studies conducted on male rats, knock down of SRY resulted in dopamine deficiency causing abnormal motor function. Reversal of the knock down led to normal SRY levels with, resumption of normal ...

AADC deficiency results in the inability to synthesize dopamine, a neurotransmitter essential for motor function and leads to ...