In 2016, the spinal muscular atrophy community celebrated the approval of the first-ever treatment that targets the underlying genetic cause of SMA. This was a milestone more than 100 years in the making, made possible by dedicated researchers, and the community that supported their work. The Discovery of SMA SMA was first described by a […]
The Discovery of SMA Read More »
Roche has provided the following community statement on RG7916 and Olesoxime investigational programs. Dear SMA Community, We are very pleased to share an update of the Roche SMA program We have two investigational molecules in development for people with SMA, olesoxime and RG7916. Both molecules are given by mouth (or g-tube) and work in different
Roche Releases Community Statement on RG7916 and Olesoxime Programs Read More »
Cure SMA has awarded a $75,000 research grant to Robert Kalb, MD, at Northwestern University Feinberg School of Medicine, for his project, “Effects of diminished SMN on segmental spinal cord innervation of motor neurons.” Dr. Kalb and his team have conducted studies that indicate that there are abnormal connections to motor neurons in their worm
Cure SMA Awards $75,000 Grant to Robert Kalb, MD, Northwestern University Read More »
Clinicians across the country are invited to share experiences, successes and ideas at the inaugural SMA Clinical Care Meeting to be held on Saturday, June 16, 2018 at the 2018 Annual SMA Conference in Dallas, Texas. As Spinraza and future FDA-approved treatments extend the lives of those with SMA, care has become even more important. Our
Cure SMA to Host Inaugural Clinical Care Meeting Read More »
Cure SMA has awarded a $150,000 research grant to Umrao Monani, PhD, at Columbia University, for his project, “SMA modulators as a means to revealing disease mechanisms.” Dr. Monani and his team have generated SMA model mice and shown that their genetic background can alter disease severity. By studying these mice, they have identified two
Cure SMA Awards $150,000 Grant to Umrao Monani, PhD, Columbia University Read More »
Biogen recently announced new interim Phase 2 results from NURTURE, the ongoing open-label, single-arm study evaluating the efficacy and safety of SPINRAZA or nusinersen among pre-symptomatic infants with spinal muscular atrophy. Additionally, data from a case series conducted on SPINRAZA-treated teens and young adults was also released. The main findings of which are summarized below:
Biogen Announces New Interim Phase 2 Results From NURTURE Read More »
Dear Members of the SMA Community, For the past several years, we’ve been working to collect data and information on our community’s experiences, goals, hopes, and challenges. We know that the voice of our community is powerful. By sharing our stories, we can communicate our priorities to the FDA and regulators, provide insight into daily
Last night, Governor Eric Holcomb of Indiana signed HB 1017, adding spinal muscular atrophy (SMA) and severe combined immunodeficiency (SCID) to the state’s newborn screening panel. The bill makes Indiana the fourth state in the country to adopt permanent SMA screening, following Missouri, Utah and Minnesota. The legislation was sponsored by Representative Doug Gutwein, and
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Cure SMA has awarded a $150,000 research grant to Allison Ebert, PhD, at the Medical College of Wisconsin, for her project, “Role of astrocyte-produced miR-146a in SMA pathology.” SMA is caused by mutations in the SMN1 gene resulting in low levels of SMN protein, which is expressed in every cell in the body. The goal
Cure SMA Awards $150,000 Grant to Allison Ebert, PhD, Medical College of Wisconsin Read More »
When a loved one is affected by spinal muscular atrophy, whether recently diagnosed or living with SMA for a while, you want to make a difference in their life and in the lives of all those affected by SMA. At Cure SMA, we believe in the power of the SMA community working together. That’s why
Awareness is Best Raised When We Work Together Read More »
