Rescindo Therapeutics’ RSC-57 receives FDA orphan drug designation and rare paediatric disease designation for Kabuki syndrome

Rescindo Therapeutics

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.

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Michael Wonder

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Michael Wonder