Shots:
The US FDA has approved Isembyld for the treatment of spinal muscular atrophy (SMA) in pts (≥2yrs.) who are currently receiving a SMN2-targeted treatment
Approval was backed by the P-III (SAPPHIRE), which met its 1EP of 2.2-point improvement in HFMSE with Isembyld + SMN2-targeted therapy vs PBO at 1yr. in main efficacy population (n=103);…
