Washington, D.C. — The Friedreich's Ataxia Research Alliance (FARA) has awarded a $300,000 grant to Edison Pharmaceuticals in the first step of an ongoing collaboration with the Muscular Dystrophy Association (MDA), and Seek A Miracle (SAM), intended to move promising compounds for the treatment of neuromuscular diseases from the lab to the clinic.
The initial FARA grant is to fund the pre-clinical toxicology and pharmacology on clinical candidates developed by Guy Miller, MD, PhD and the team of Edison Pharmaceuticals of San Jose, California.
"FARA is very excited to work with MDA and SAM on this project," said Ron Bartek, FARA’s president. "We believe these compounds show real promise in addressing the respiratory chain defect present in Friedreich’s ataxia patients. Our hope is that clinical candidates such as Edison’s EPI-A0001 will form the basis of a first-line treatment in arresting the progression of FA."
"It is so encouraging for Seek A Miracle and MDA to collaborate with FARA on a project that shows so much promise for treating our disease," said Samantha Litke-Wilson, president of Seek A Miracle MDA.
There is no effective treatment or cure available for Friedreich's ataxia. FA is a hereditary disorder that often first becomes noticeable as a balance and coordination problem in children of elementary school age. Further loss of strength and coordination in all four extremities usually forces those with FA into wheelchairs by their teens. FA is progressive and can lead to impaired vision, speech and hearing. FA is also commonly associated with severe heart disease, spinal abnormalities and diabetes. In the later stages of the disorder, patients are frequently incapacitated with cardiac failure leading to death. Friedreich's ataxia, although rare, is the most prevalent inherited ataxia, affecting about 1 in every 50,000 people in the United States.
The Friedreich’s Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's Ataxia (FA). It was founded by FA affected families and scientists in 1998. Since then, it has provided almost $3 million in grants to scientists around the world who are pursuing treatments. http://www.FAResearchAlliance.org
Seek A Miracle (SAM) is dedicated to raising funds for MDA’s research in Friedreich’s ataxia. The collaboration has raised more than $1.3 million since 1994. Initially, MDA will earmark $100,000 from Seek A Miracle specifically for the collaborative effort to develop drugs for human trials. http://www.seekamiracle.org
Muscular Dystrophy Association (MDA) is a voluntary health agency working to defeat more than 40 neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education. http://www.mdausa.org
President, Friedreich’s Ataxia Research Alliance