Cure SMA Awards $150,000 Grant to Charlotte Sumner, MD, Johns Hopkins University

Leave a Comment / About SMA, Front Page News, Research /

Cure SMA has awarded a $150,000 research grant to Charlotte Sumner, MD, at Johns Hopkins University, for her project, “Testing the potential of SMN-AS1 as a therapeutic target in SMA.” Dr. Sumner and her team have recently identified a long noncoding RNA (lncRNA) that represses the ability of SMN2 to make its template for protein […]

Cure SMA Awards $150,000 Grant to Charlotte Sumner, MD, Johns Hopkins University Read More »

Record Number of Abstracts Submitted for 2018 SMA Researcher Meeting

Leave a Comment / Conference, Front Page News, Research /

Abstract submission is now closed for the 2018 SMA Researcher Meeting. We are excited to announce that we received 128 submissions, a record number. Our submissions come from researchers in 16 countries, 11 companies, and close to 50 institutions. We expect over 400 researchers and clinicians from almost 100 organizations will attend the SMA Researcher

Record Number of Abstracts Submitted for 2018 SMA Researcher Meeting Read More »

Roche Releases Community Statement on RG7916 and Olesoxime Programs

Leave a Comment / Clinical Trials, Front Page News, Research /

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 Awards $75,000 Grant to Robert Kalb, MD, Northwestern University

Leave a Comment / Front Page News, Our Impact, Research /

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 »

Cure SMA to Host Inaugural Clinical Care Meeting

Leave a Comment / Conference, Front Page News, Support & Care /

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 Awards $150,000 Grant to Umrao Monani, PhD, Columbia University

Leave a Comment / Front Page News, Our Impact, Research /

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 Announces New Interim Phase 2 Results From NURTURE

Leave a Comment / Clinical Trials, Front Page News, Research /

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 »

Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment

Leave a Comment / Community Awareness, Front Page News, Research /

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

Cure SMA Launches Second Annual Community Survey to Address Important Issues in SMA Treatment Read More »

Indiana is the Fourth State to Adopt Permanent SMA Screening

Leave a Comment / Advocacy, Community Awareness, Front Page News /

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

Indiana is the Fourth State to Adopt Permanent SMA Screening Read More »

Scroll to Top