2023 Volume 40 Issue 5 Pages 712-716
Motor neuron diseases (MND) are devastating neurodegenerative disorder which primary affects motor neurons : amyotrophic lateral sclerosis (ALS), spinal bulbar muscular atrophy (SBMA), and spinal muscular atrophy (SMA). In 2022, results of phase 3 trial of ultra–high dose methylcobalamin (JETALS) and tofersen, an antisense nucleotide for SOD1 mutation (VALOR) were published. Ultra–high dose methylcobalamine was effective for ALS–patient 1–year within the onset. VALOR trial did not reach the primary endpoint, although open label extension trial suggested the positive efficacy.
This review provides an overview of clinical advances in MND research and summarizes selected key literature on therapeutic approaches in 2022.
