Methylmalonic acidemia (MMA) is a rare and potentially fatal genetic disorder that affects metabolism and multiple body systems that affects about one in 100,000 infants. It's caused by a genetic ...

LB-001 is under clinical development by Alexion Pharmaceuticals and currently in Phase II for Methylmalonic Acidemia. According to GlobalData, Phase II drugs for Methylmalonic Acidemia does not have ...

isovalemic acidemia; LCHADD, long chain 3-chydroxy-acyl-CoA dehydrogenase deficiency; MCADD, medium chain acyl-CoA dehydrogenase deficiency; MMA, methylmalonic acidemia; NAGS, N-acetylglutamate ...

mRNA-3705 is under development for the treatment of methylmalonic acidemia (MMA). It acts by targeting methylmalonyl CoA mutase (MUT) enzyme. The therapeutic candidate comprises of messenger RNA (mRNA ...

A baby’s alarming deterioration was the result of a disorder that can be overlooked in infants, and that, if left untreated, ...

A new year means a new Fierce Biotech Fundraising Tracker to record all the venture capital being funneled into the industry for 2024. We're bumping up our reporting criteria from last year's ...

The gastroenterologist also ordered a test to check for methylmalonic acidemia, a rare genetic disorder present at birth that impairs the breakdown of fat and protein and can cause feeding ...

1 Amgen R&D Postdoctoral Fellows Program, Amgen Inc, South San Francisco, CA, United States 2 Amgen Research, Amgen Inc., South San Francisco, CA, United States Tolerogenic vaccines represent a ...

Introduction: Gestational Diabetes Mellitus (GDM) is a metabolic disorder marked by Q10 hyperglycemia that can negatively affect both mothers and newborns. The increasing prevalence of GDM and the ...