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 ...

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 ...

PTC Therapeutics announced the initiation of a Phase 2 trial of ataluren to treat patients with nonsense mutation methylmalonic acidemia (nmMMA). MMA is a rare genetic disorder caused by deficiencies ...

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 ...

Researchers have discovered a hormone in a mouse study that can be used immediately to can help doctors predict how severely patients with the rare disease methylmalonic acidemia are affected and when ...

New Rochelle, NY, April 1, 2016--A new study in patients with the inherited metabolic disorder methylmalonic academia (MMA) found lower than expected levels of antibodies against the adeno-associated ...

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WATERTOWN, Mass., and Research Triangle Park, N.C., Oct. 20, 2020 (GLOBE NEWSWIRE) -- Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc ...

Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, has announced the first patient has been dosed in the Phase 1/2 study ...

(RTTNews) - Moderna Inc. (MRNA) said that the first patient has been enrolled in the Phase 1/2 study evaluating the safety and tolerability of mRNA-3705. mRNA-3705 is an investigational mRNA ...

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 / ...