New Drug Treats Disfiguring Genetic Disorder

A Maryland 10-year-old is one of the first patients in the state to be treated with a new drug for a rare genetic disorder that can be disfiguring and painful.

The drug, called Cinryze, was approved in October by the Food and Drug Administration and is proving to be quite successful.

Nicholas Moronta and his mother, Jeanette, made the trip from Montgomery County to the Asthma, Allergy and Sinus Center in Rosedale to see Dr. Manov Singla.

Singla was instrumental in getting the new drug approved that treats a painful rare genetic disease from which both of the Morontas suffer called hereditary angioedema. It affects about one in every 50,000 people.

The disease happens when a protein that's missing in the blood leads to severe swelling. Sometimes, sufferers' faces are unrecognizable, doctors said.

While Jeanette Moronta said she doesn't have attacks very often, Nicholas' case is acute. He has suffered severe abdominal swelling and attacks in his larynx that close off his airway.

His mother said previous treatments couldn't be used safely in children and caused horrible side effects, but with Cinryze, she said he has a chance at a normal childhood.

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