Friday, February 26, 2016

What are the Treatments for Rare Diseases?

  • According to the Kakkis EveryLife Foundation, 95% of rare diseases have not one single FDA approved drug treatment.
  • During the first 25 years of the Orphan Drug Act (passed in 1983), only 326 new drugs were approved by the FDA and brought to market for all rare disease patients combined.
  • For many patients with rare diseases, there is no specialist.  Doctors simply treat the symptoms of the disease.  For most, this means seeing multiple specialists to address various needs. This is the same for Abby.  The closest we have to a specialist with CCMS is Dr. C., who is the only person to ever see what CCMS ribs look like.  She sees specialists for pulmonary (2!), GI, ophthalmology, orthopedics, and cardiology.  When we add therapies, Abby sees 11 different people to address symptoms related to her rare disease.

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