- In February 2022, Asklepios BioPharmaceutical, Inc., a subsidiary of Bayer AG, announced that the European Commission (EC) granted orphan drug designation to AB-1003, also referred to as LION-101, for treating limb girdle muscular dystrophy. This designation highlights the drug's potential in addressing the needs of patients with this rare condition. Securing orphan drug status can facilitate expedited development and regulatory processes, paving the way for earlier access to much-needed therapies
- In September 2024, Atamyo Therapeutics has collaborated with the Dion Foundation for Children with Rare Diseases to initiate a clinical trial in the U.S. for ATA-200, a promising gene therapy aimed at treating limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). This partnership highlights a commitment to advancing innovative therapies for patients affected by this rare condition. The trial's expansion into the U.S. signifies a crucial step in exploring the potential of ATA-200 as a treatment option for individuals suffering from LGMD2C/R5
