This new analysis of 5-year data on a novel gene therapy from PTC Therapeutics shows both cognitive and motor function improvement in children with aromatic L-amino acid decarboxylase (AADC) ...

The study portrays the intense need for daily care experienced by children with aromatic L-Amino acid decarboxylase deficiency (AADC-d) and their families. Parents or caregivers of children with the ...

The FDA granted accelerated approval to eladocagene exuparvovec (Kebilidi) to treat children and adults with aromatic L-amino acid decarboxylase (AADC) deficiency, the agency announced Thursday. The ...

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 ...

Five-year-old Boston Yannotti’s ear-to-ear smile masks what has been an extremely difficult childhood. The young boy, with reddish-brown hair and bright blue eyes like his mother, has been confined ...

What Is Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency? AADC deficiency is an ultra-rare genetic disorder. It affects how your cells send signals to each other. People born with this condition ...

WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics (PTCT), Inc. announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC ...

Texas Children's is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency. Aromatic l-amino acid ...

SAN DIEGO and CAMBRIDGE, Mass., March 28, 2019 (GLOBE NEWSWIRE) -- Neurocrine Biosciences, Inc. (NASDAQ:NBIX) and Voyager Therapeutics, Inc. (NASDAQ:VYGR) today announced the publication of interim ...

SOUTH PLAINFIELD, N.J., May 17, 2021 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced data from the first study on the impact of caring for a child with the ultra-rare genetic ...