In spinal muscular atrophy linked to SMN1 (SMA), a single dose of a novel gene therapy called GC101 improved motor function in nine patients with type II and type III SMA aged six months to 18 years.

In this Research Highlights issue, we present recent study findings on thymectomy and CAR-T cell therapy in myasthenia, as well as the evaluation of ChatGPT for supporting rehabilitation in adolescents with myositis.

Antwerp put the spotlight on CMT by hosting the second European congress dedicated to Charcot-Marie-Tooth disease. The event strengthened cooperation across Europe among researchers, clinicians and patients. Discover the key highlights: promising treatments and diagnostic innovations.