This year, Rare Disease Day occurs on February 29—the rarest day of all! Spinal muscular atrophy (SMA) affects 12,000 people in the U.S. Yet, the SMA community is part of a greater community—the rare disease community—that comprises of 300 million people worldwide. Together, we create an even stronger voice to raise awareness and educate others […]
Celebrate Rare Disease Day 2020 with Cure SMA Read More »
Genentech, a member of the Roche Group, today presented 1-year data from the pivotal Part 2 of the SUNFISH study, evaluating risdiplam in people aged 2-25 years with Type 2 or Type 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure 32 scale
Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today highlighted key accomplishments of the past year and announced R&D priorities for 2020. Below please find a community statement from Scholar Rock specifically discussing SRK-015. ______________________________________________________________________________ Dear SMA Community, We at Scholar
Community Statement from Scholar Rock Regarding SRK-015 Read More »
Elizabeth and Brian Veit live in Jefferson City, Mo., with their two children—Benjamin, who is 6 years old, and Kate, who is 5 years old. By all accounts, Elizabeth’s second pregnancy was “completely normal,” and the health and vitality of her first child created little reason for concern. But when Elizbeth and Brian started noticing
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