Cure SMA has awarded a $140,000 research grant to Chad Heatwole, MD, at the University of Rochester for his project, “Development of a Clinically Relevant Outcome Measure for SMA Therapeutic Trials.” As more spinal muscular atrophy drug programs progress to clinical trials, it becomes critically important to develop ways of accurately measuring whether or not […]
The Annual SMA Conference consists of two separate events that run in parallel. The first is focused on families affected by SMA, who gather for workshops, keynote sessions, networking, and fun events such as a dance party, movie night, and more. The second event is the SMA Researcher Meeting, the largest research meeting in the
SMA Researcher Meeting to Host Over 100 Presentations Read More »
Biogen, in collaboration with Isis Pharmaceuticals, has announced that they will soon open recruiting for NURTURE. This is a multi-center, Phase 2 clinical study evaluating the efficacy of the investigational drug, ISIS-SMNRx (ISIS 396443), in pre-symptomatic newborns that have a genetic diagnosis of Spinal Muscular Atrophy (SMA). The purpose of the NURTURE study is to
Biogen Announces NURTURE, a New Phase 2 Clinical Trial Read More »
On Monday, February 9, 2015, Newfield Exploration presented Cure SMA with a grant for $2,500 to support ongoing research in finding a cure. Accepting the grant on behalf of Cure SMA were Marynell Larson, president of the Cure SMA Rocky Mountain Chapter, and Michelle Pritekel, board member of the Rocky Mountain Chapter. In 2001, Newfield
Newfield Exploration Gives Generous Grant to Cure SMA Read More »
Cure SMA is pleased to announce that we have extended our spinal muscular atrophy research program with the California Institute of Biomedical Research (CALIBR) with an additional $315,000 drug discovery grant. This partnership first began in 2012, when we awarded $700,000 to Dr. Peter G. Schultz and his team for their drug discovery program, “Optimization
When Cure SMA first started funding research thirty years ago, we had to build up from virtually nothing. It’s no easy task. Drug development relies on volume to be successful—just one or two options are not enough. The vast majority of drug candidates end in failure. To arrive at just one FDA-approved drug for SMA,
Cure SMA Releases Updated Overview of Research Model Read More »
The Annual SMA Conference consists of two separate events that run in parallel. The first is focused on families affected by SMA, who gather for workshops, keynote sessions, networking, and fun events such as a dance party, movie night, and more. The goal is to lend each other support and strength while learning about the
Cure SMA Hosts SMA Researcher Meeting this June Read More »
Spinal muscular atrophy is the number one genetic cause of death for infants, and 1 in 50 people carry the genetic mutation that causes SMA. But even so, many people are not familiar with SMA. Thanks to our community, that is changing. Melissa Joan Hart recently posted about SMA awareness on her Instagram and Twitter
How Our Cure SMA Community is Raising Awareness Read More »
When our year-end campaign began in mid-November, we set an initial goal of raising $540,000. Our community quickly met that challenge, so we increased our goal to $750,000—the most ever raised as part of our annual campaign. We surpassed that goal with over $770,000 by the end of December. So now we’re setting our sights
Cure SMA Annual Campaign Sets $1 Million Goal Read More »
Over the past decade, one of the most important advances in SMA drug development has been the number of pharmaceutical companies investing in treatments for SMA. Ten years ago, we had just one company. Now, we have a dozen. These additional companies bring additional resources and expertise that will get us to treatments and a
Updates on Pharmaceutical Companies Investing in SMA Drug Development Read More »
