12 February 2021 - Rescindo Therapeutics announced today that the U.S. FDA has granted orphan drug designation and rare paediatric disease designation for the Company’s lead drug RSC-57, for the treatment of patients with Kabuki Syndrome.
Kabuki Syndrome is a rare developmental disorder characterised by intellectual disability, muscle hypotonia, distinctive facial features, short stature, immune deficiency and malformations of the cardiac, renal, gastrointestinal, and/or skeletal systems.