Kerala doctors successfully performed a life-saving liver transplant on a two-and-a-half-year-old child diagnosed with the ...
Metabolic cardiomyopathy is an important diagnostic consideration in a young patient with unexplained ventricular dysfunction or sudden cardiac arrest. Propionic acidemia is an uncommon but important ...
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 ...
Metabolic disorders and organic acidemias comprise a diverse group of inherited conditions in which enzymatic deficiencies disrupt normal metabolic pathways. Such deficiencies lead to the accumulation ...
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 ...
PALO ALTO, Calif., Aug. 18, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and ...
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