Pharmacothérapie des maladies neuromusculaires : ce qui a changé en 2025
Abstract
Two new drugs have recently become available for treating neuromuscular diseases: omaveloxolone and tofersen. Omaveloxone, which is available in Switzerland, mitigates mitochondrial oxidative stress and has been shown to slow the clinical progression of Friedreich's ataxia. Tofersen is an antisense oligonucleotide that induces posttranscriptional silencing of the SOD1 (superoxide dismutase 1) gene, which is involved in familial forms of amyotrophic lateral sclerosis. Although its efficacy has so far only been indirectly demonstrated through its ability to reduce serum neurofilament levels, it is available on a compassionate use basis in Switzerland.
Fields of Study & Subjects
Amyotrophic Lateral Sclerosis Research
Neurogenetic and Muscular Disorders Research
Genetic Neurodegenerative Diseases
Publication details
- Journal / source
- JMIG
- Volume
- 22
- Issue
- 945
- Pages
- 66–70
- Publication date
- 1 January 2026
- Language
- EN