Galactosemia is a rare genetic condition that prevents babies from processing galactose, an important sugar in breast milk. Though the disease can cause many issues, it’s easily diagnosed and ...

Galactosemia is a rare genetic disorder that causes an inability to digest galactose, which is a breakdown product of lactose. Lactose is one of the main carbohydrates in milk. In the infant ...

Galactosemia is a rare genetic, metabolic disorder caused by a deficiency in the galactose-1-phosphate uridyltransferase (GALT). This means patients cannot break down galactose, ...

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

The Galactosemia Foundation, a non-profit charitable organization that advocates for people with Galactosemia and their families, issued the following statement in response to the U.S. Food and ...

The phase 3 ACTION-Galactosemia Kids trial evaluated govorestat vs placebo in children (n=47) aged 2 to 17 years with classic galactosemia. The primary endpoint was the global statistical test ...

Shares of Applied Therapeutics APLT surged 68.8% on Wednesday after it announced a regulatory update regarding the ongoing new drug application (NDA) review of APLT’s lead candidate, govorestat ...

In the phase III ACTION-Galactosemia Kids study in children with galactosemia aged 2-17 years, govorestat improved daily activities, behavior, cognition, fine motor skills and tremor.

Summary. Applied Therapeutics is a clinical-stage biotech focused on treating symptoms of galactosemia in the central nervous system. Govorestat (AT-007) is in late-stage regulatory approval for ...

ACTION-Galactosemia Kids was designed to evaluate the impact of govorestat vs. placebo on clinical outcomes over time in 47 children aged 2–17 with galactosemia.

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 ...

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 ...