What’s Up Doc? Gene therapy research offers hope for methylmalonic acidemia patients

Q: Is there a new treatment being developed to cure methylmalonic acidemia?A: Methylmalonic acidemia, also known as MMA, is an inherited autosomal recessive condition — the patient must get a defective gene from each of their parents. MMA patients are unable to break down certain fats and proteins, causing a buildup of methylmalonic acid in their blood. Although MMA encompasses a spectrum of disorders, with different mutations causing different [...]
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