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) ...

“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 ...

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 ...

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 ...

AADC deficiency is a rare and fatal genetic disorder that typically causes severe disability starting during the first months of life. Children living with AADC deficiency can experience seizure-like ...

Logan Lacy has spent his 4.5 years trapped in a body controlled by a newborn's nervous system. He has never run or climbed like kids his age, let alone sit up on his own or speak. But his parents, ...

MOBILE, Ala. (WALA) -A mother brings attention to a rare life-threatening disease. Kelly Heger is living proof of the length parents will go to take care of a child with an ultra-rare disease. In ...

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 ...