“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” said Levi Garraway, M.D., Ph.D., Genentech’s Chief Medical Officer and Head of Global Product Development. “The Evrysdi tablet combines established efficacy with convenience, providing an […]
To address the chronic health needs of individuals with spinal muscular atrophy (SMA), Cure SMA and the SMA community have launched a new advocacy campaign in support of federal research for SMA. Through the campaign, Cure SMA seeks dedicated SMA research funding through a federal research program managed by the U.S. Department of Defense (DOD).
Cure SMA Launches Advocacy Campaign to Secure New Federal Research for SMA Read More »
February marks Rare Disease Month, a time to amplify the voices of the 300 million people worldwide living with a rare disease—including the individuals in the U.S. impacted by spinal muscular atrophy (SMA). Throughout this month, and especially during Rare Disease Week (starting February 23rd), we’re committed to advancing research, raising awareness, and advocating for
Rare Disease Month 2025: Driving Progress Through Research Read More »
