Silver SpringThe FDA approved the first Sanfilippo treatment Thursday.
Sanfilippo syndrome destroys children’s brains and kills before adulthood, with no prior approved drug.
Called Fayuvi, the Ultragenyx therapy replaces the missing enzyme whose absence lets toxic sugar build up in cells.
Ultragenyx has not disclosed a price for the ultra-rare disease treatment.
How each outlet framed it
- STAT leans favorable
- centers patient/family perspective, quoting foundation chief on shift from 'take child home' to treatment hope
Sources: STAT