The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have decreased AADC activity that causes severe disability, including gross motor ...

AADC deficiency is an ultra-rare genetic disorder that affects how your cells send signals to each other. Learn more about this disorder's symptoms, causes, diagnosis, and treatment.

The FDA's accelerated approval is based on the safety and clinical efficacy findings from the ongoing PTC-AADC-GT-002 global clinical trial.

The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency.

The FDA has granted accelerated approval to Kebilidi (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with AADC deficiency.

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

The FDA has approved PTC Therapeutics’ Kebillidi (eladocagene exuparvovec-tneq) for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. According ...

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

AADC deficiency is caused by a mutation in the gene that produces the AADC enzyme, this enzyme is needed to produce a neurotransmitter called dopamine which is important in controlling movement.

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

AADC is an enzyme involved in dopamine and serotonin synthesis. Eladocagene exuparvovec is a recombinant adeno-associated virus serotype 2-based therapy that delivers a functioning human DDC gene ...