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Families of Spinal Muscular Atrophy Receives FDA Orphan Drug Designation For Quinazoline495 For The Treatment of Spinal Muscular Atrophy
Date:8/28/2009

e Orphan Drug Act facilitates a close working relationship between regulatory agencies and companies aimed at accelerating the drug development and approval processes for treatment of rare diseases. Since the Act was passed in 1983, the Food and Drug Administration (FDA) has approved more than 200 new orphan products.

For further information, please see: http://www.fda.gov/orphan/oda.html

About Families of Spinal Muscular Atrophy:

Families of Spinal Muscular Atrophy is dedicated to creating a treatment and cure by:

  • Funding and advancing a comprehensive research program;
  • Supporting SMA families through networking, information and services;
  • Improving care for all SMA patients;
  • Educating health professionals and the public about SMA;
  • Enlisting government support for SMA;
  • Embracing all touched by SMA in a caring community.

Our vision is a world where Spinal Muscular Atrophy is treatable and curable.

A small group of parents started Families of SMA in 1984. They wanted to raise funds for SMA research to cure the disease, and support all affected families. Back then, very little was known about Spinal Muscular Atrophy. Very little research was being conducted. No one knew the cause of the disease let alone how to find a treatment and a cure. There were no family support services and no clinical trials. Patients and families affected by SMA were on their own and had little hope.

Today, FSMA has a different story to tell. Families of SMA has created hope for the SMA community that did not exist in 1984. FSMA has raised and funded over $50 million for SMA research. Support comes from generous individual donations and numerous fundraising events held by volunteer families an
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SOURCE Families of Spinal Muscular Atrophy
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