Galactosemia is an inherited genetic disorder of metabolism where the body is unable to metabolize a sugar called galactose. Galactose forms half of the sugar lactose, which is found in milk. The ...

It’s easy to test for galactosemia. It’s also simple to treat once it’s diagnosed. And children who have it can live normal lives. Type I occurs in about 1 in every 30,000 to 60,000 people.

The US Food and Drug Administration (FDA) has declined to approve Applied Therapeutics’ govorestat for classic galactosemia, ...

Nearly three months after an experimental treatment for classic galactosemia was rejected by the US Food and Drug ...

Galactosemia is a rare genetic disorder that results from the body’s inability to properly metabolize galactose, converting instead to the toxic metabolite, galactitol, which is known to cause ...

Investors reading into the U.S. FDA’s decision to skip an advisory committee meeting to discuss the NDA for Applied Therapeutics Inc.’s govorestat, seeking approval as the first therapy for treating ...

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 ...

9 advisory committee meeting to review its lead pipeline candidate, the classic galactosemia treatment govorestat, ahead of a planned November decision on approval. By canceling its planned ...