The Millers’ three-year-old son, Evan, exhibited frequent symptoms of distress. “People kept telling us he was just a cranky, colicky baby,” says Amanda. At 12 weeks of age, Evan abruptly stopped taking any food and screamed when anyone touched his abdomen. In the emergency room, it was determined that Evan had fluid in his abdomen and low blood sugar. His blood wasn’t clotting and his kidneys and spleen were enlarged. Most alarming, there were signs of liver failure.
The Children’s Hospital of Philadelphia diagnosed the condition as tyrosinemia type 1, which is a rare metabolic disease.
The orphan drug Orfadin® was approved by FDA in 2002. However, because Jon is self-employed as an auto mechanic, the family did not have insurance that would cover the cost of the drug. Jon connected online with another parent who told him about NORD and its Patient Assistance Programs, which help patients obtain needed medications. While other organizations and companies provide PAPs for medications today, NORD was the first to do so.
NORD partners with Rare Disease Therapeutics on a PAP for Orfadin®. When Jon Miller called to inquire about that program, he spoke to Michelle Mortara, a patient assistance specialist. NORD, the specialty pharmacy, and the company providing the drug.
Within one day, the medication arrived at the hospital, and little Evan and his parents were soon on their way home. Today, Evan attends pre-school and “does all the things three-year-olds like to do,” his mother says.
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