ELK GROVE VILLAGE, Ill., Sept. 2, 2014 /PRNewswire-USNewswire/ -- Families of SMA, founded in 1984 by a small group of families affected by spinal muscular atrophy (SMA), has announced that the organization will become Cure SMA. Spinal muscular atrophy is a motor neuron disease that affects approximately 1 in every 10,000 births. The most common form of SMA is often fatal in the first two years of life, making SMA the leading genetic cause of death for infants.
With 15 drug programs underway, involving collaborations with a dozen of the largest pharmaceutical and biotech companies, a treatment and cure for SMA appears closer than ever before. Cure SMA has provided funding for two-thirds of those drug programs, contributing over $55 million to SMA research. The organization recently announced an additional $1.8 million commitment to research over the next six months.
"This change comes at a time of great promise for our community," said Kenneth Hobby, president of Cure SMA. "Particularly over the last decade, we've seen tremendous progress in SMA research. We hope to accelerate that momentum as we advance towards a FDA-approved treatment for SMA"
"Unlike many 'orphan diseases,' where research can languish for years, the outlook for SMA is bright," said Jill Jarecki, PhD, Cure SMA's research director. "We've identified the genetic cause of SMA, and discovered a 'back-up gene' already in the body, which is a promising target for new drugs. However, that is just one of several treatment avenues we are pursuing."
Cure SMA also provides support to families affected by SMA—reaching nearly 4,000 in the last year, including two-thirds of all newly diagnosed families in the US—and hosts the largest SMA conference in the world each June. The conference joins researchers and families, so they can learn from and collaborate with each other.
"Our families will continue to be the focus of everything we do," said Hobby. "With new supporters, pharmaceutical and research partners—including governmental and regulatory agencies—joining our community over the last decade, this is the right moment to open our doors to all who are passionate about developing a cure for SMA."
About Cure SMA
Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)—a disease that takes away a person's ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.
Since 1984, we've directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA. We are currently on the verge of breakthroughs in treatment that will strengthen our children's bodies, extend life, and lead to a cure.
We have deep expertise in every aspect of SMA—from the day-to-day realities to the nuances of care options—and until we have a cure, we'll do everything we can to support children and families affected by the disease.
Learn more about how you can help us reach a treatment and cure at www.cureSMA.org.
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SOURCE Cure SMA
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