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
|SOURCE Families of Spinal Muscular Atrophy|
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