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 ...
isovalemic acidemia; LCHADD, long chain 3-chydroxy-acyl-CoA dehydrogenase deficiency; MCADD, medium chain acyl-CoA dehydrogenase deficiency; MMA, methylmalonic acidemia; NAGS, N-acetylglutamate ...
These are Maple Syrup Urine Disease, Methylmalonic Acidemia/Propionic Acidemia, Galactosemia, Phenylketonuria, Gaucher Disease, Pompe Disease, Fabry Disease, MPS II (Hunter Syndrome), MPS IV ...
A baby’s alarming deterioration was the result of a disorder that can be overlooked in infants, and that, if left untreated, ...
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 ...
Among the diseases included are maple syrup urine disease, Methylmalonic acidemia/propionic acidemia, galactosemia, phenylketonuria, gaucher disease, pompe disease, fabry disease, Hunter syndrome ...
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 ...
Philippine Health Insurance Corporation (PhilHealth) announced on Monday new benefit packages covering various illnesses as part of its commitment in the implementation of the Universal Health Care ...
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 ...
There are some exceptions to this concept of “placental protection”; for example, disorders that affect energy metabolism, such as the primary lactic acidoses and glutaric aciduria type II or ...
Fetal Medicine Research Unit, University of Bristol, Department of Obstetrics, St Michael’s Hospital, Bristol BS2 8EG Professor Peter Soothill. If you wish to reuse ...