A new publication with our clinical collaborators at Columbia University Medical Center in New York reveals sensory-motor circuit dysfunction in human Spinal Muscular Atrophy (SMA) patients as was predicted by our Drosophila model of SMA. Unfortunately 4-AP treatment, which we found was efficacious in models to correct sensory-motor deficits, at lower levels does not seem to produce significant locomotion benefits for SMA patients. Lower doses must be employed in patients to avoid undesirable side effects.
