Double Blind Study in Phase #2 with Mestinon (pyridostigmine) in patients with spinal muscular atrophy types 2–4

Placebo-controlled, double-blind cross-over trial with pyridostigmine and placebo to investigate the effect and efficacy of pyridostigmine on muscle strength and fatigability in patients with genetically confirmed SMA. We aim to include 45 patients with SMA types 2–4, aged 12 years and older in the Netherlands. Participants receive 8 weeks of… Att fortsätt läsa