• The Never-ending Quest to Gain Weight
  • #AAN2018 — Spinraza Improves Motor and Respiratory Function in SMA Children, Trials Show
  • #AAN2018: SMA Treatment Candidate RG7916 Increases Amount of Key SMN Protein, Clinical Trials Show
  • A Message for Us
  • Adventures in the Big Apple (Part 1)
  • #AAN2018 – Olesoxime Stabilizes SMA Patients’ Long-term Movement Function, Extension Study Shows
  • For My Best Friend, My Brother
  • Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
  • An Update on My Spinraza Journey
  • NIH Agency Pioneers Collaborative Research into Rare Diseases
  • ‘Family Perspectives’: A Reflection on the Interviews
  • Muscle Waste Product Creatinine Might Be Used as SBMA Biomarker, Study Reports