SMA Press

The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that the FDA has approved a label extension for Evrysdi(risdiplam) to include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that pre-symptomatic babies treated with Evrysdi for at least one…

Today Scholar Rock shared a community update on SAPPHIRE, a clinical trial evaluating apitegromab in non-ambulatory patients with Type 2 and 3 SMA: Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of apitegromab in those living with Spinal Muscular…

Roche recently shared an update on MANATEE with the SMA community: Update on Roche’s SMA global clinical development programme: New global combination study to begin in early 2022 Dear members of SMA Europe, In response to your request, we are pleased to share with you the initiation of MANATEE, a new global Phase 2/3 clinical…

August is SMA Awareness Month. To mark the occasion, Karen Chen, CEO of the SMA Foundation, was featured in several interviews where she discussed SMA, the recent FDA drug approvals SMA, and the remaining unmet need for patients. Please see below for links to the interviews. https://empoweredpatientradio.com/accelerating-development-of-treatments-for-spinal-muscular-atrophy-with-karen-chen-sma-foundation

Scholar Rock announced positive six-month interim analysis results from the TOPAZ Phase 2 clinical trial yesterday. SRK-015 is a selective inhibitor of myostatin that has been shown to increase muscle mass and strength in animal models. Treatment with SRK-015 led to improvements in Hammersmith scale scores (primary efficacy endpoint) in all three cohorts of patients…