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 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 ...
Keypoint: The appellate court ruled that the California Age-Appropriate Design Code Act’s impact assessment provision is unconstitutional and remanded the case back to the trial court to consider the ...
(RTTNews) - PTC Therapeutics Inc. (PTCT) presented a new analysis of five-year results that shows its gene therapy, PTC-AADC, leads to profound improvements in children with aromatic L-Amino acid ...
On Thursday, the FDA granted accelerated approval to PTC Therapeutics, Inc.’s (NASDAQ:PTCT) gene therapy for AADC deficiency, the first-ever gene therapy approved in the U.S. that is directly ...
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 ...
“This is nothing short of a miracle," mom Shante Stagg told TODAY of a gene therapy that has helped her son who has a rare health condition. After a complicated birth, Jamell Stagg Jr.’s parents ...
In March 2023, a young girl came to Boston Children’s Hospital unable to hold up her head — one striking symptom of aromatic L-amino acid decarboxylase (AADC) deficiency. This rare neurological ...
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