Latest News
Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)
Genentech, announced earlier this month new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at […]
Read More ›SMA Community Seeks SSI Improvements on Program’s 50th Anniversary
Fifty years ago today, the Supplemental Security Income (SSI) program was created to “provide supplemental security income to individuals who have attained age 65 or are blind or disabled” for […]
Read More ›New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA
Scholar Rock yesterday announced new quality-of-life (QoL) data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment which indicate stabilization or continued improvement […]
Read More ›Community Spotlight: Amber-Joi Watkins and Family
The Igbo and Yoruba proverb, “It takes a village to raise a child” has been in use for centuries and remains in use across languages and cultures due to its […]
Read More ›Cure SMA Holds Patient-Led Listening Session with FDA
On Thursday, August 4, 2022, six members of the SMA community had the opportunity to talk directly with the U.S. Food and Drug Administration (FDA) about their lived experiences with […]
Read More ›A Deliciously Easy Way to Fundraise for Cure SMA
Nothing brings people together more than sharing a meal, but what if you could make that meal even more meaningful? We’re encouraging all members of our SMA community to consider […]
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