February 2020

Grant Announcement: Cure SMA Awards $150,000 Grant to Dmytro Morderer, Ph.D., Mayo Clinic, Jacksonville, FL

This article is part of a series of Cure SMA grant announcements that will be shared throughout the next couple of months. Cure SMA has awarded a $150,000 grant to Dmytro Morderer, Ph.D., of the Mayo Clinic in Jacksonville, Fla., for his project titled, “Effect of SMN deficiency on ribonucleoprotein assembly.” Dr. Morderer is a […]

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Grant Announcement: Cure SMA Awards a $170,000 Grant to Lyndsay Murray, Ph.D., University of Edinburgh

This article is part of a series of Cure SMA grant announcements that will be shared throughout the next couple of months. Cure SMA has awarded a $170,000 research grant to Lyndsay Murray, Ph.D., at the University of Edinburgh, for her project titled, “Developing strategies to support enlarged motor units following SMN restoration in mouse

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Cure SMA Working Group Revises Recommendations for the Treatment of Infants Diagnosed with SMA via Newborn Screening Who Have 4 Copies of SMN2

To help clinicians and the families they serve in the decision of when to administer therapy to infants identified with SMA via newborn screening, Cure SMA convened a working group comprised of 15 SMA experts to develop treatment guidelines. These guidelines, “Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening,” were originally

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Walk-n-Roll with Cure SMA in 2020!

It is that time of year again…time to walk and roll with Cure SMA. In 2020, we will host nearly 50 Cure SMA Walk-n-Rolls in partnership with our committed volunteers across the country, raising more than $1.7 million! These walk events are family-friendly and bring together walkers, rollers, volunteers, and local/national partners to raise money

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Cure SMA Launches “Spotlight on SMA” Partnership with Neurology Reviews

Cure SMA is pleased to announce the release of “Spotlight on SMA: The Urgent Need for Early Diagnosis in Spinal Muscular Atrophy,” a supplement developed in collaboration with Neurology Reviews. The goal of this partnership is to enhance current awareness and understanding of SMA diagnostic requirements, while emphasizing the importance of early treatment as it

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Celebrate Rare Disease Day 2020 with Cure SMA

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

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Lights…Camera…Action! Search Is On for Child with SMA to Appear in NBC Television Pilot

Cure SMA is excited to support casting directors from NBC as they search for boys and girls from the SMA community who are interested in playing a character with spinal muscular atrophy in an upcoming 20th Century Fox pilot. The team is currently focusing their search on children aged 7-11 years of age in the Chicagoland

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Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 Years with Type 2 or Type 3 SMA

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

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