Galactosemia is a group of inherited metabolic disorders characterized by the body’s inability to produce a sufficient amount of enzymes to process galactose. The mostly commonly insufficient enzymes are GALT, GALK, and GALE. Patients are identified at birth through newborn screening (NBS) and are immediately put on a restricted diet, which is the only available treatment. However, dietary restriction does not always prevent long-term complications. Galactosemia is characterized by a varied phenotypic spectrum, ranging from normal development to severe complications, such as jaundice, cataracts, failure to thrive, bacterial sepsis, learning difficulties, neurological impairment, primary ovarian insufficiency (POI), and liver and kidney disease. Some therapies with novel mechanisms of action are in early clinical development and, if efficacious, stand to capitalize on the immense unmet need for effective therapies in this space.
3 KOL interviews in May 2020
KEY COMPANIES COVERED
Applied Therapeutics, Moderna Therapeutics
KEY DRUGS COVERED
AT-007, GALT mRNA, salubrinal, GALK1 inhibitors
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