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Antisense Morpholino Treatment to Increase SMN Levels and Treat Spinal Muscular Atrophy
TS-037648 — A treatment for spinal muscular atrophy.
Spinal Muscular Atrophy (SMA) is caused by deletion or mutation of SMN1 and retention of SMN2, this in turn leads to reduced levels of the full SMN protein. SMA is the second leading cause of neuromuscular disease, and many people living with SMA need round-the-clock care from their families and a…
  • College: College of Medicine (COM)
  • Inventors: Burghes, Arthur; Porensky, Paul
  • Licensing Officer: Flammang, Ann Marie

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