Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action
The purpose of this study is to assess whether 4-AP (Dalfampridine-ER, Ampyra) improves walking ability and endurance in adult patients with Spinal muscular atrophy (SMA) Type 3 compared to placebo and whether the duration of treatment affects outcome.
• Aged 18 to 50 years at the time of enrollment
• Have genetically confirmed SMA 3 (homozygous absence of SMN1 exon 7)
• Ability to walk at least 25 meters without assistance
• Be free of major orthopedic deformities (i.e. scoliosis, contractures)
• Normal Cystatin C clearance (> 80 ml/min)