Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

Exclusion Criteria

• •1. Non-ambulatory type 3 adults and all type 2 adults.
• •2. Patients with co-morbid conditions that preclude travel, testing or study medications.
• •3. Patients who have participated in a treatment trial for SMA in the 3 months prior to this trial, or plan on enrolling in any other treatment trial during the duration of this trial.
• •4. Patients who are, in the investigator's opinion, mentally or legally incapacitated from providing informed consent for the study, or are otherwise unable to meet study requirements or cooperate reliably with study procedures, especially strength testing.
• •5. Patients with a need for non-invasive ventilatory support (e.g. BiPAP) for \> 12 hours/day
• •6. Transaminases, amylase or lipase \> 3.0 x normal values, WBC \< 3.0 or neutropenia \< 1.0, platelet count \< 100 K, or hematocrit \< 30 persisting over a 30 day period
• •7. Use of medications or supplements which interfere with VPA metabolism and increase the potential risks of the medications, or are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders within 3 months of study enrollment. These agents include riluzole, creatine, butyrate derivatives, growth hormone, anabolic steroids, daily albuterol use, anticonvulsants, or other HDAC inhibitors.
• •8. Women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding.