Current paediatric standard-of-care for galactosemia is a galactose-free diet. Image credit: Shutterstock / Photoongraphy. Nearly three months after an experimental treatment for classic galactosemia ...

Current paediatric standard-of-care for galactosemia is a lactose-free diet. Image credit: Shutterstock / Photoongraphy. Nearly three months after an experimental treatment for classic galactosemia ...

The Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to Applied Therapeutics regarding the New Drug Application (NDA) for govorestat for the treatment of classic ...

The NDA included data from the phase 1/2 ACTION-Galactosemia study (ClinicalTrial.gov Identifier: NCT04117711) and the phase 3 ACTION-Galactosemia Kids trial (ClinicalTrial.gov Identifier ...

The US Food and Drug Administration (FDA) has declined to approve Applied Therapeutics’ govorestat for classic galactosemia, prolonging the wait for the first marketed treatment for the genetic ...

There are around 3,300 patients in the US with galactosemia, an inherited metabolic disorder that affects the body’s ability to properly convert galactose to glucose. A deficiency in the enzyme ...

announced that the FDA has issued a Complete Response Letter regarding their New Drug Application for govorestat, a treatment for Classic Galactosemia. The letter indicates that the FDA found ...

Galactosemia Foundation Urges FDA to Reconsider on Behalf of Rare Disease Community Facing Devastating Disease ALBANY, N.Y., Nov. 27, 2024 /PRNewswire/ -- The Galactosemia Foundation, a non-profit ...

Galactosemia affects approximately 3,300 patients in the U.S. and 4,400 in the E.U., with mandatory newborn screening in these regions. Currently, there are no treatment options for this ...