Advisory Board March 2, 2023
FDA on Tuesday approved a new treatment for Friedreich’s ataxia, a rare and debilitating disease, using data collected from patient communities over decades, Amy Dockser Marcus writes for the Wall Street Journal — a move that opens a potential pathway for other rare disease treatments to gain approval.
FDA approves new treatment for Friedreich’s ataxia
Friedreich’s ataxia is a neurological disorder that causes progressive damage to the spinal cord, muscle weakness, and movement problems. It often kills those who have it by the age of 35. Currently, there are only around 22,000 people with the condition worldwide, with most being in the United States, Europe, and Australia.
On Tuesday, FDA approved Reata Pharmaceutical’s drug omaveloxolone or Skyclarys to treat Friedreich’s...
Today's Sponsors
Today's Sponsor