Scholar Rock today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead antibody product candidate, SRK-015, for the treatment of spinal muscular atrophy (SMA). SRK-015 is an inhibitor of the activation of myostatin, which Scholar Rock believes has the potential to be the first muscle-directed therapy […]
Cure SMA has awarded a $150,000 research grant to Robin Parks, PhD, at the University of Ottawa, for his project, “Serum-derived exosomes as a biomarker for Spinal Muscular Atrophy.” Dr. Parks and his team are exploring the use of exosomes, small particles released from all cells, as potential biomarkers for SMA. They have shown that
Cure SMA Awards $150,000 Grant to Robin Parks, PhD, University of Ottawa Read More »
Cure SMA is thrilled to welcome Biogen back as a National Premier Partner for 2018. As a part of their partnership, Biogen will be sponsoring all walk-n-rolls and galas across the US for the entirety of 2018, as well as the Hope on the Hill Congressional Dinner. Biogen is also a key participant in many
Cure SMA Welcomes Back Biogen as a National Premier Partner for 2018 Read More »
Today, Congress passed a new federal spending bill, increasing funding for several critical newborn screening programs, that the President also signed. The bill increases funding for the Health Resources and Services Administration (HRSA) Heritable Disorders Program by more than $2 million, and increases funding for the CDC’s newborn screening quality assurance program by almost $4 million. The
House and Senate Vote to Increase Federal Funding for Newborn Screening Programs Read More »
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 »
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 »
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 »
