Science Daily

New discovery offers real hope for rare genetic disease

Scientists discovered that certain gene changes allow cells to function even when frataxin, the protein lost in Friedreich’s ...
STAT

In a first, a nonprofit wins FDA approval for rare disease gene therapy

The FDA has granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...
Fierce Pharma

Italian nonprofit lands FDA approval for 1st gene therapy to treat rare genetic disease

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...
BioSpace

FDA Greenlights First Gene Therapy for Rare Pediatric Disease

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...