SMA Community Progress & Advocacy Featured at Cure SMA Hope on the Hill Event

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Congressional leaders and key partners, supporters, and members of the spinal muscular atrophy (SMA) community celebrated SMA achievements and advocacy at Cure SMA’s 11th Annual Hope on the Hill Congressional Event. Hope on the Hill is an annual event to increase awareness about SMA and recognize policymakers and other key partners for helping to advance […]

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Cure SMA to Host 11th Annual Hope on the Hill Event in Washington, D.C.

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On December 6th, Cure SMA will highlight the progress made in spinal muscular atrophy (SMA) and recognize the advocacy that helped lead to this success in a special event in our nation’s capital. Cure SMA has hosted an annual Hope on the Hill congressional dinner and reception in Washington, D.C. since 2010 to increase awareness

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Community Spotlight: Michelle Tynski and Family

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Play is important for kids. It allows children to develop cognitive, social, emotional, and physical skills while engaging creatively. But it’s not just how kids play that’s important, what they play with also matters. Which is why Michelle Tynski, mom to nine-year-old Trace, Benjamin who passed away in 2016, and 3-year-old Zach, who has Type

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Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA)

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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 time of enrollment. Patients had been previously treated with other approved or investigational SMA-targeting therapies, including Spinraza® or Zolgensma®. Data showed Evrysdi improved or maintained motor function

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SMA Community Seeks SSI Improvements on Program’s 50th Anniversary

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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 their basic living needs, such as housing, clothing, medical costs, and food. The program and its monthly cash benefit have been a lifeline for many

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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

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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 with apitegromab for nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) receiving an SMN-targeted therapy. The TOPAZ trial assessed activities of daily

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Community Spotlight: Amber-Joi Watkins and Family

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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 continued relevance. For Amber-Joi Watkins, her husband, Tommy Domalski, and their daughter, Céline, that village includes family and friends, as well as Céline’s pediatrician, her

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Cure SMA Holds Patient-Led Listening Session with FDA

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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 SMA and their unmet medical needs. This Patient-Led Listening Session was a private teleconference attended by patient advocates, FDA representatives from the Center for Drug

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