Past Grants Awarded by Fight SMA

2008

International Spinal Muscular Atrophy Patient Registry
The SMA Patient Registry is supported by the Patient Advisory Group of the International Coordinating Committee for SMA Clinical Trials which includes: Families of SMA, FightSMA, Muscular Dystrophy Association, SMA Foundation, and other SMA advocacy groups.

Evaluation of compounds that increase SMN protein levels
Elliot Androphy, University of Massachusetts Medical School

Restoration of SMN Levels in Mild SMA Mice Using Lentiviral Gene Therapy
Matthew Butchbach, Ohio State University

Testing SMN inducing compounds in SMA mice
Chris Lorson, University of Missouri Columbia

Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants with Spinal Muscular Atrophy (STOP SMA)
Kathy Swoboda, University of Utah Medical School

2007

Impact of In-Exsufflator Treatment on Hospitalization for Respiratory Exacerbation in Neuromuscular Disease
Sherri Katz and Ian MacLusky, Childrens’ Hospital of Eastern Ontario Research Institute

Pre-Symptomatic Infants With Spinal Muscular Atrophy
Kathy Swoboda, University of Utah Medical School

Autonomic Dysfunction in Childhood Onset Spinal Muscular Atrophy
John R. Bach, University of Medicine and Dentistry of New Jersey

Novel SMN interacting proteins- novel targets for treatment of SMA

Elliott Androphy, University of Massachusetts

Research to modulate aberrant termination events in SMA
Ellen Welch, PTC Therapeutics, Inc.

A Large Animal Model of Spinal Muscular Atrophy
Monique Lorson, University of Missouri Columbia

2006

Autonomic Dysfunction in Childhood Onset Spinal Muscular Atrophy

John R. Bach, University of Medicine and Dentistry of New Jersey

Miniature Wireless Motion and Position Sensors
Nigel Livingston, University of Victoria

In silico analysis of SMN inducing pathways
Alex MacKenzie, Children’s Hospital of Eastern Ontario

SFHR-mediated modification of murine ES-derived SMA motor neurons: A step forward through a novel cell therapy for Spinal Muscular Atrophy
Federica Sangiuolo, Tor Vergata University School of Medicine, Rome, Italy