- In March 2025, ThermoGenix Biocare announced the launch of a next-generation copper histidinate injectable formulation designed specifically for enhanced bioavailability and pediatric safety in Menkes Syndrome patients, marking a significant advancement in copper replacement therapy.
- In February 2025, RareGene Therapeutics Inc. received Orphan Drug Designation from the U.S. FDA for its investigational gene therapy candidate RGT-501, targeting ATP7A mutations associated with Menkes Syndrome, supporting further clinical development and regulatory incentives.
