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About SMA: 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…
Read MoreToday 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…
Read MoreRoche 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…
Read MoreAugust 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
Read MoreThe Spinal Muscular Atrophy (SMA) Foundation and PTC Therapeutics have entered a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders. Recent FDA approvals of several disease modifying therapies for SMA has made regenerative medicine approaches the next frontier in drug discovery and development. The SMA Foundation-PTC…
Read MoreScholar 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…
Read MoreFollowing the approval of Evrysdi, Roche released an article describing its partnership with the SMA Foundation and PTC Therapeutics to develop the drug. To read the article, please visit: https://www.roche.com/partnering/spinal-muscular-atrophy-sma-together.htm
Read MoreArya Singh, an SMA patient and Yale University student, was featured in a New Haven Register article on September 19th. In the article, Singh describes the challenges she faces as a patient and her experiences in clinical trials. To view the article, please visit: https://www.ctinsider.com/news/nhregister/article/This-Yale-student-won-t-let-a-rare-genetic-15579131.php?src=nhrhpcp To view a photo essay for the article, please visit:…
Read MoreIn August, SMA News Today published a series of articles on the discovery and development of Evrysdi, the first and only oral medicine for SMA approved by the FDA. The articles cover a range of topics including the role of the SMA Foundation in drug development, the experiences of patients taking Evrysdi, and the future…
Read MoreAs part of its “Collaborations That Transform” webinar series, WuXi AppTec hosted three panels of distinguished SMA experts, representing patient, industry and physician perspectives. The panelists discussed the current state of SMA therapies and the promise of the next generation of treatment approaches. To view the webinar please visit: https://wxpress.wuxiapptec.com/webinar-sma/
Read MoreThe SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that Evrysdi™ (risdiplam) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of Spinal Muscular Atrophy (SMA) in adults and children two months and older. Evrysdi showed clinically meaningful improvements in motor function across two clinical trials covering a broad range…
Read MoreThe SMA Foundation’s partners, Roche and PTC Therapeutics, announced positive data from the pivotal Part 2 of FIREFISH, a study evaluating risdiplam in infants aged 1-7 months with Type 1 SMA. The study met its primary endpoint of a statistically significant proportion of infants sitting without support for at least five seconds after one year…
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