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 ...

We sought to predict renal growth based on clinical and metabolic parameters in patients with isolated methylmalonic acidemia, a group of disorders associated with chronic kidney disease. Fifty ...

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 ...

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 / ...

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 ...

RESEARCH TRIANGLE PARK, N.C. and WATERTOWN, Mass., Nov. 19, 2020 (GLOBE NEWSWIRE) -- Asklepios BioPharmaceutical, Inc. (AskBio) and Selecta Biosciences, Inc. (NASDAQ: SELB) today announced that the ...

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 ...

The FDA has placed Logicbio Therapeutics Inc.’s phase I/II clinical trial of LB-001, an investigational AAV genome-editing therapy for treating pediatric patients with methylmalonic acidemia (MMA), on ...

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 ...

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/ ...

(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 ...