Author name: Sarah McCall

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

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 Read More »

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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A Deliciously Easy Way to Fundraise for Cure SMA

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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 hosting a dining fundraiser, where a portion of purchases are designated to support Cure SMA. This is also a great way to supplement your efforts

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Special NBS Awareness Month Announcement: South Carolina Screens for SMA!

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Cure SMA is pleased to announce that South Carolina is now screening babies born in the state for spinal muscular atrophy (SMA). South Carolina Department of Health and Environmental Control (DHEC) officials shared the exciting news with Cure SMA in a written statement earlier today: Good news! The new multiplex PCR based method for screening

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Kids: We Invite You to Bake a Difference for Cure SMA!

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Cure SMA is calling all future bakers and pastry chefs to help us bake a difference for individuals with spinal muscular atrophy (SMA). Baking a Difference was created by kids for kids with sweet hearts and a love of sweet treats. From a red wagon selling treats outside a local grocery store to full-fledged enterprise

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Seat Elevators on Power Wheelchairs are a Necessity, not a Luxury, according to the SMA Community

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Individuals with spinal muscular atrophy (SMA) who utilize power wheelchairs for their mobility agree that seat elevation, which allows an individual to adjust their wheelchair seat, helps improve mental and physical health, increase quality of life, and promote independence. The SMA community’s views were shared in a public comment letter Cure SMA sent to the

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Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks

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Cure SMA is conducting a survey to understand our community’s perspectives on treatment possibilities and tolerance for the side effects of currently available and planned therapies. The results will help inform our future objectives and priorities as well as help us continue to bring the patient voice to regulatory authorities. Some of you may recall

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