News

Gene Therapy Candidate Found to Provide Clinical Benefits for Up to 5 Years

PTC Therapeutics‘ gene therapy candidate PTC-AADC (formerly AGIL-AADC) provided clinically meaningful and sustained improvements in motor, cognitive, and language milestones in children with aromatic l-amino acid decarboxylase (AADC) deficiency up to five years following the one-time treatment, trial analyses show. A single dose of PTC-AADC delivered into…

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…