A new treatment may be able to treat a life-threatening disorder that predominantly affects children, initial findings from a pioneering clinical trial suggest.
There is currently no cure for PA. Instead, treatments aim to manage the symptoms of the disease, by getting patients to follow a carefully controlled, low-protein diet, for example, and frequently replacing lost fluids to prevent dehydration. Patients may also require liver transplants, as replacing this key organ involved in metabolism can help somewhat restore their levels of functioning PCC.
By submitting your information you agree to the Terms & Conditions and Privacy Policy and are aged 16 or over.The patients were split into five groups, and each group was given injections of varying amounts of mRNA-3927 over a few weeks to see what dose of the treatment worked best with the fewest side effects.
"This research could potentially improve outcomes for patients with propionic acidemia, who are at risk for recurrent illnesses requiring hospitalization, impaired growth and development, and early mortality," Dr. Dwight Koeberl, a professor of pediatrics at Duke University School of Medicine in North Carolina who was not involved in the research, told Live Science in an email.
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