Families of Spinal Muscular Atrophy Announces Latest Developments on Three SMA Drug Discover... (gin clinical trials was completed in Oct...)

Families of Spinal Muscular Atrophy Announces Latest Developments on Three SMA Drug Discovery Programs

Date:6/18/2009

gin clinical trials, was completed in October 2008. According to Chris Airriess, Ph.D., Chief Operating Officer, California Stem Cell, Inc., "CSC is now preparing for a final FDA pre-IND meeting to take place in mid 2009, keeping us on track for a formal IND application to begin a Phase I/IIA clinical trial in SMA Type I."

Tetracycline Program:

According to Paul Higgins, Ph.D., Director, Inflammation Drug Discovery, Paratek Pharmaceuticals, "FSMA has supported the research at Paratek for the previous three years for our work on tetracycline derivatives as a potential treatment of SMA. These compounds are intended to correct SMN2 splicing and in turn increase SMN protein levels.

Using funding from FSMA to generate the preliminary data for a grant application, Paratek has been awarded a multi-million dollar U01 grant from the NINDS to continue our research that FSMA funded for the past 3 years."

The overall goal for this project is to develop a drug candidate for SMA resulting in an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) within 4 to 5 years.

These three announcements were all made at the 25th Anniversary SMA Conference. A record of over 900 families and researchers were present at the conference.

About Families of SMA:

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.

The goal of the organization for 2009 is to have raised $50 million for SMA r
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Families of Spinal Muscular Atrophy Announces Latest Developments on Three SMA Drug Discover...(gin clinical trials was completed in Oct...),Health