2009 Families of SMA Conference

Post date: May 17, 2013 2:22:48 PM

2009 Families of SMA Conference

Highlights of this years conference included:

-HBO filming a documentary during the conference.

-Keynotes speeches given at the main Conference Banquet from:

Paula Barrett Chair of the Board of Directors of Families of SMA.

Dr. Jill Jarecki. Research Director Families of SMA.

The Honorable Steve L. Driehaus. US House of Representatives.

Dr. Hans Keirstead. World famous stem cell scientist and Co-Director 

of the Sue and Bill Gross Stem Cell Research Center and Associate Professor of Anatomy and Neurobiology at the University of California Irvine.

Dr. Mary Schroth. Leading pediatric pulmonologist at the University of 

Wisconsin American Family Children's Hospital. She also chairs the FSMA 

Medical Advisory Council and is a researcher with Project Cure SMA.

The latest Developments on three drug discovery programs funded by Families of Spinal Muscular Atrophy were announced at the Conference.

1) Quinazolines to boost SMN2 expression;

2) Tetracyclines at Paratek Pharmaceuticals to correct SMN2 splicing;

3) Motor neuron replacement program at California Stem Cell and UCI.

These three programs represent an investment of over $16 Million so far to build a SMA drug pipeline.

S. 1158 SMA Treatment Acceleration Act

S. 1158, The SMA Treatment Acceleration Act of 2009

To authorize the Secretary of Health and Human Services to conduct activities to rapidly advance treatments for spinal muscular atrophy, neuromuscular disease, and other pediatric diseases, and for other purposes. This Bill was introduced in THE SENATE OF THE UNITED STATES OF AMERICA, on May 21, 2009 by Ms. Stabenow, Mr. Isakson and Mr. Whitehouse. The Bill was read twice and referred to the Committee on Health, Education, Labor, and Pensions

H.R.2149 SMA Treatment Acceleration Act

H.R. 2149, The SMA Treatment Acceleration Act of 2009

To authorize the Secretary of Health and Human Services to conduct activities to rapidly advance treatments for spinal muscular atrophy, neuromuscular disease, and other pediatric diseases, and for other purposes. This Bill was introduced in THE HOUSE OF REPRESENTATIVES on April 28, 2009 by Mr. Kennedy and Mr. Cantor. The Bill was referred to the Committee on Energy and Commerce.

SMA Treatment Acceleration Act of 2009 - Amends the Public Health Service Act to require the Director of the National Institutes of Health (NIH) to establish a national clinical trials network for spinal muscular atrophy by upgrading and unifying spinal muscular atrophy clinical trial sites and recruiting new investigators and sites.

Requires the Director to ensure that such network:    

(1) Conduct coordinated, multisite, clinical trials of therapies and clinical approaches to the treatment of Spinal Muscular Atrophy.

(2) Rapidly and efficiently disseminates scientific findings to the field.