The second AADC Deficiency Awareness Day will take place virtually on Oct. 22. It is sponsored by the…
Articles by Forest Ray PhD
A gene therapy dramatically improved the motor functions and quality of life in children with aromatic L-amino acid decarboxylase…
Examining cerebrospinal fluid — the liquid surrounding the brain and spinal cord — remains the best way to diagnose…
Diagnosing aromatic L-amino acid decarboxylase (AADC) deficiency in newborns is “feasible” using dried blood spots and readily available equipment, allowing…
The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected…
PTC Therapeutics is preparing to launch PTC-AADC, its experimental gene therapy for aromatic l-amino acid decarboxylase (AADC) deficiency,…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the…
The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure…
The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during…