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Project Cure SMA is a collaborative initiative between Families of SMA and clinical investigators designed to help facilitate the rapid translation of promising new therapies to individuals with SMA. The primary goal of Project Cure SMA is to develop safe and well-tolerated clinical protocols to help identify effective therapies for SMA.

• All 6 six study sites are actively recruiting study patients
• Utah study site has completed their patient enrollment for both study cohorts
• We are anticipating completion of study patient recruitment by the end of September 2006
• Our goal is to be able to publish the study results by early 2008

• A modified Hamersmith functional motor scale for use in multi-center research on SMA

The study group needing to fill a few available slots is looking for patients age 2 to 8 at the time of study enrollment, SMA 2 or non-ambulatory SMA 3. All subjects must be able to sit independently for at least 3 seconds without support.

There are exclusions to this study including, but not limited to: the need for BiPAP support > 12 hours per day; Spinal rod or fixation for scoliosis or anticipated need within six months of enrollment; Use of medications or supplements which interfere with valproic acid or carnitine metabolism within 3 months of study enrollment; Current use of either VPA or carnitine; Body Mass Index > 90th % for age; Coexisting medical conditions that contraindicate travel, testing or study medications

In the second group we are looking for patients age 3-17 at the time of study enrollment, SMA types 2 or 3, who can stand independently without braces or other support for up to 2 seconds, or walk independently.

The exclusions for this group include, but are not limited to: Spinal rod or fixation for scoliosis or anticipated need within six months of enrollment; Current use of either VPA or carnitine; Body Mass Index > 90th % for age; Use of medications or supplements which interfere with valproic acid or carnitine metabolism within 3 months of study enrollment; Coexisting medical conditions that contraindicate travel, testing or study medications; Transaminases, amylase or lipase > 3.0 x normal values, WBC < 3.0 or neutropenia < 1.0, platelets < 100 K, or hematocrit < 30 persisting over a 30 day period.

If you are interested in participating and meet the above criteria, please contact the SLC Project Cure Site (see Locations.) You may also call the Project Cure Clinical Trial Manager, Dr. Sandra Reyna at 801-581-3551 or sreyna@projectcuresma.org