Cure SMA is excited to announce that AveXis is returning as a National Premier Partner for 2018. As part of their partnership, AveXis is generously supporting our Chapter Leader Summit, a full day of training and information-sharing for 70 chapter leaders from around the US, scheduled for April 2018. Many of the topics and training […]
Cure SMA Welcomes Back AveXis as a 2018 National Premier Partner Read More »
Cure SMA has awarded a $150,000 research grant to Brunhilde Wirth, PhD, at Institute of Cologne, Germany, for her project, “Study of combinatorial therapy based on SPINRAZA together with a novel protective genetic modifier.” Based on so-called SMA discordant families, in whom SMN1-deleted individuals remain asymptomatic, Dr. Wirth and her team have recently identified two
Cure SMA Awards $150,000 Grant to Brunhilde Wirth, PhD, Institute of Cologne, Germany Read More »
Cure SMA has awarded a $50,000 research grant to Rashmi Kothary, PhD, at the University of Ottawa, for his project, ” The shifting landscape of SMA research: towards a better understanding for a role for SMN in aging.” In this proposal, Dr. Kothary and his team seek to explore the role of SMN protein in
Cure SMA Awards $50,000 Grant to Rashmi Kothary, PhD, University of Ottawa Read More »
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 »
