Science Day Session 6: SMA Therapies and Real-World Evidence

Fri, 2 October, 10:30-12:00

Chairs:

  • Blood biomarkers for longitudinal monitoring in adult 5q spinal muscular atrophy (SMA) (C. Stapf, Halle)
  • Metabolic alterations in Spinal Muscular Atrophy (C. Herrmann, Ulm)
  • Long-term trajectories of multimdimensional fatigue in adults with 5q-spinal muscular atrophy receiving disease-modifying therapy (Bogdan Bjelica, Hannover)
  • Biomarker potential of established and exploratory serum muscle parameters in adult patients with SMA before and during treatment with nusinersen and risdiplam (H. Lapp, Dresden)
  • Challenges and opportunities of current and future SMA therapies (T. Hagenacker, Essen)

Event Timeslots (1)

Room 63, 53, 43
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