The Hill News

Having a seriously ill child is always difficult and painful. It’s even more so when a child’s illness is one of the 7,000 rare diseases for which cures and treatments are hard to find.

Rare diseases affect more than 15 million children in the United States.  A disease is considered rare if it affects less than 200,000 people; however the majority of rare diseases are considered ‘ultra rare’ meaning that they affect less than 6,500 patients.  Diseases such as Cystic Fibrosis, Muscular Dystrophy, Huntington’s disease and pediatric cancers are some of the better known rare diseases that strike children and adults.  But there are thousands more that touch only a few hundred lives and are virtually unknown to the broader public.

Because these diseases are rare, support networks are hard to find and information can be difficult to obtain. Even getting a diagnosis can be a challenge because doctors may not know what they’re looking for. An! d when the diagnosis comes, it is often followed by the frustrating news that no cure exists and treatment options are minimal. 

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