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Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Clinical Trial in SMA
Date:5/19/2011

ies for the treatment of SMA to date, which affects one in every 6,000 babies. One in every 40 people carry the gene that causes SMA, indicating approximately 7.5 million carriers in the United States.

One of the goals at FSMA is to fund early stage drug discovery programs for SMA to the point where other groups will invest.  At the earliest stages of drug development, programs have less than a 1% chance of success.  This inherent risk along with low potential for profit due to a small patient population has traditionally hindered industry from working on orphan diseases.  

FSMA has actively reduced the barriers to SMA drug discovery programs by providing:  1) early seed funding, 2) access to tools and reagents, 3) expert SMA advisors, and 4) established clinical trial protocols and networks.

FSMA recently released a Request for Proposals for new preclinical drug discovery programs to build a robust drug pipeline for SMA.  Partnerships such as this one with Repligen, between non-profits, the government and companies are a very effective way to share the risks of developing rare disease treatments.  This FSMA approach also enables the correct expertise for a particular stage of development to be brought into a program.

About Families of Spinal Muscular Atrophy: Families of SMA is a non-profit 501(c)3 tax exempt organization with 30 Chapters throughout the United States and over 70,000 members and supporters. 

Families of SMA funds and directs the leading SMA research programs.  The successful results and progress from basic research to drug discovery programs to clinical trials provide real hope for families and patients.

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 he
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SOURCE Families of Spinal Muscular Atrophy
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