The law, which went into effect at signing, contains significant design and development requirements, requires independent ...

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 ...

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) ...

Eladocagene exuparvovec is the first US-approved gene therapy for AADC deficiency, administered directly to the brain. Clinical trials showed significant motor and cognitive improvements, especially ...

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 ...

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, ...

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

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 ...

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 ...