Chair, NINDS Spinal Muscular Atrophy Development Project. Created a virtual discovery company (medicinal chemistry, in vitro and in vivo biology, adme, safety) on this multi-year, multi-million dollar project that is targeting a disease modifying treatment for SMA. Project is at the candidate selection / Pre-IND stage with a stop codon readthrough compound.
Co-chair of NINDS Translational Research Evaluation Committee