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 ...

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 ...

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 ...

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 ...

CAMBRIDGE, MA / ACCESSWIRE / April 3, 2024 / Moderna, Inc. (NASDAQ:MRNA) today announced that interim data for a first-in-human, phase 1/2, open-label, dose optimization study and extension study, ...

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 ...

mRNA-3927 uses the same proprietary LNP formulation as mRNA-1944 (antibody against Chikungunya virus) and mRNA-3704 (methylmalonic acidemia) “Fast Track designation underscores the urgent need for a ...

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 ...