Methylmalonic acidemia and cobalamin deficiencies represent a group of inborn errors of metabolism that arise from impaired processing of vitamin B12 (cobalamin). This impairment often results in the ...

Methylmalonic acid (MMA) is a substance that is created when your body digests protein. The amount of vitamin B12 in your body controls how much MMA your body makes. A high amount of MMA typically ...

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 ...

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering ...

Center for Biologics Evaluation and Research has chosen mRNA-3705 as one of four investigational medicines for accelerated development to address unmet medical needs for rare diseases CAMBRIDGE, MA / ...

Genomic methods, specifically whole exome and whole genome sequencing, have been at the center of diagnosing rare, genetic diseases. For example, diagnosing the inborn errors of metabolism (IEM) ...

Deficiencies can occur when you don’t get enough B-12 from your diet or when your body can’t use it properly. Typically, vitamin B-12 deficiencies can be detected through the vitamin B-12 test.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced the first patient ...

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mRNA-3704 is Moderna’s first rare disease program to enter clinical studies CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering ...