Spinal muscular atrophy

Additional Resources
https://treat-nmd.org/treat-nmd-diseases/spinal-muscular-atrophy/
https://www.musculardystrophyuk.org/
https://rarediseases.org/rare-diseases/spinal-muscular-atrophy/
Revision history
This material was produced by Dr Michela Guglieri at the John Walton Muscular Dystrophy Research Centre, Newcastle University, UK and Dr Mariacristina Scoto MD, PhD, Consultant in Neuromuscular Translational Research, Honorary Lecturer, The Dubowitz Neuromuscular Unit, UCL Great Ormond Street Institute of Child Health, UK. Additional support was provided by Avril Palmeri, John Walton Muscular Dystrophy Research Centre.
The material has been reviewed by Sheonad Macfarlane, mother of a child with SMA; Daniel Kostan, father of a child with SMA, Vice-president of SMAci - SMA Patient organization; Stephen Sayer, SMA adult patient and Dawn Wilbur, SMA Type 3 adult patient.