The National Institute of Health (NIH) has created a new approach to diagnosing and treating methylmalonic acidemia (MMA), a rare genetic disorder that stops the body from metabolizing fats and…
When gene therapy company Asklepios BioPharmaceutical, Inc. ("AskBio") and biotechnology company Selecta Biosciences ("Selecta") joined forces to combat genetic disorders, each company brought its best technology and insights. AskBio…
Fast Track Designation and Rare Pediatric Disease Designation have just been awarded from the FDA to HemoShear Therapeutics. This designation was given to their new investigative therapy for propionic acidemia…
Methylmalonic Acidemia Methylmalonic acidemia (MMA) is a rare disease affecting the metabolic system. 60% of all cases are due to a deficiency in MUT, a mitochondrial enzyme, which causes acids to…
The National Institutes of Health (NIH) is the primary medical research institution in the United States. The NIH is one of several public health institutions, like the Food and Drug…
Methylmalonic Acidemia (MMA) is a liver condition which affects the body's ability to break down food and fatty acids. A dietary imbalance or minor virus for these patients may spark a metabolic…
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