CatalystFDA/Bio

Ultragenyx wins FDA approval for pediatric gene therapy for Sanfilippo syndrome

FDA approval grants Ultragenyx a new pediatric gene therapy for Sanfilippo syndrome.

What changed

FDA approval grants Ultragenyx a new pediatric gene therapy for Sanfilippo syndrome.

Why it matters

FDA approval grants Ultragenyx a new pediatric gene therapy for Sanfilippo syndrome. — impact context not separately stored; see What changed.

Sources