- In January 2025, Dynacure presented Phase I/II study results for DYN101, its antisense drug candidate for CNM, at the World Muscle Society conference.
- In February 2025, Astellas launched a CNM clinical trial registry platform to consolidate global patient participation across centers.
- In March 2025, Biogen signed a strategic agreement with Genethon for preclinical CNM gene therapy development.
- In April 2025, Ionis Pharmaceuticals initiated a global trial evaluating systemic administration of a next-gen antisense platform for rare myopathies.
