Kerala doctors successfully performed a life-saving liver transplant on a two-and-a-half-year-old child diagnosed with the ...

Cholesterol bloodstream The FDA has granted Fast Track designation to mRNA-3927 (Moderna), an investigational mRNA therapeutic for propionic acidemia. The Food and Drug Administration (FDA) has ...

The Food and Drug Administration (FDA) has approved a new indication for Carbaglu ® (carglumic acid; Recordati Rare Diseases) for use as adjunctive therapy in the treatment of acute hyperammonemia due ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of ...

The Institute for Quality and Efficiency in Health Care (IQWiG) was commissioned by the Federal Joint Committee (G-BA) to investigate whether screening for vitamin B12 deficiency and the diseases ...

Moderna announced the preliminary results of a Phase I/II dose optimization and extension study to assess the efficacy and safety of mRNA-3927, an investigational mRNA treatment for propionic acidemia ...

This community project is an initiative through the University of Wisconsin Biochemical Genetics Clinic and the Wisconsin Newborn Screening Program to identify members of the Plain population who are ...

CHARLOTTESVILLE, Va., Dec. 16, 2020 /PRNewswire/ -- HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, is pleased to announce that the U.S. Food and ...

When COVID-19 hit and much of the world was forced to stay in place from spring 2020, many of us were left scared and uncertain about the future. But for parents with chronically ill children, they ...