Galactosemia is a rare genetic ... “Like lots of the inherited metal diseases, this is an enzyme deficiency. And so actually, at a biochemical level, we know exactly what the problem is ...

This enzyme deficiency leads to muscle problems and ... But that biotech is currently focusing the development of this drug for galactosemia, a different rare disease with no FDA-approved treatment.

Galactosemia (GAL), due to Gal-1-PO 4 uridyltransferase deficiency, is an inborn error of galactose metabolism causing neonatal morbidity and mortality. The general ...