Risdiplam

Unassigned

New Medicines

Spinal muscular atrophy

Information

New molecular entity
PTC Therapeutics
PTC Therapeutics

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

RG7916 may have the potential to target the underlying cause of SMA by increasing SMN protein levels in the nervous system, muscles, and other tissues by modifying the splicing of the SMN2 gene to generate more full-length SMN mRNA in SMA pts.[1]
Spinal Muscular Atrophy (SMA) affects 1 in every 11,000 children born.[1]
Spinal muscular atrophy
Oral

Evidence based evaluations