The U.S. Food and Drug Administration (FDA) has introduced a process it says will ease and speed the regulatory approval…
Articles by Lindsey Shapiro, PhD
Members of the European Parliament (MEPs) spanning party lines have called for a coordinated effort focused on improving the lives…
Note: This story was updated March 21, 2025, to correct that Pamela Price was a competing athlete in “American Ninja…
Parents of children living with rare diseases reported a range of unmet needs across multiple areas in a recent survey…
Half of aromatic l-amino acid decarboxylase (AADC) deficiency patients given the one-time gene therapy Kebilidi (eladocagene exuparvovec) exhibited…
A team of U.S. researchers developed an artificial intelligence or AI-based algorithm to help doctors decide which type of genetic…
Direct delivery of gene therapies into the central nervous system (CNS) — an approach used by Upstaza (eladocagene exuparvovec),…
Researchers have uncovered the effects of gene mutations that are associated with mild or moderate forms of aromatic L-amino…
A national research network in Canada is working to accelerate research and treatment advances for rare diseases that affect children,…
Genomics companies Ambry Genetics and PacBio are coming on board to help the GREGoR Consortium achieve its…