FDA Approves Targeted RNA-based Therapy to Treat Rare Disease

The U.S. Food and Drug Administration recently approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidoisis (hATTR) in adult patients. This rare debilitating and often fatal genetic disease is characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart and other organs. Onpattro is the first FDA-approved treatment for patients with this disease. It is also the first FDA approval of a new class of drugs called small interfering ribonucleaic acid (siRNA) treatment.

This treatment allows physicians to treat a disease by targeting the root cause, enabling them to arrest or reverse a condition rather than merely slowing its progress or treat its symptoms. RNA acts like a messenger within the body’s cells, carrying instructions from DNA for controlling the synthesis of proteins. Since its discovery in 1998, scientists have used RNA interference as a tool to investigate gene function and its involvement in health and disease.

This new class of drugs, called siRNAs, works by silencing a portion of RNA involved in causing the disease. More specifically, Onpattro encases the siRNA into a lipid nanoparticle to deliver the drug directly into the liver during an infusion treatment to alter or halt the production of disease-causing proteins.

If you suffer from a rare condition and have been denied your long-term care benefits from companies such as Unum, Hartford, or Cigna, the attorneys at Burke Harvey can help. Contact us today for a review of your case.

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