An Italian phase II, randomised, crossover, placebo-controlled trial evaluated the safety and efficacy of amifampridine, a molecule that improves neuromuscular junction function, in 13 adults with type III SMA. They were on average 34 years old, ambulant and had not previously received other innovative therapies. The results, published in June 2022, show that compared to placebo, amifampridine :
- significantly improved motor function on the FMSE test and fatigue,;
- has no significant effect on the 6-minute walk test, the ability to get up from the floor or a chair, climb stairs or walk for 10 minutes;
- is well tolerated, the main adverse effect reported being transient paresthesias.
