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New PhRMA Report: A Decade of Innovation in Rare Diseases

February 25, 2015

  • Targeted therapies can now effectively treat many of the recently identified mutated forms of CML and allow for treatment plans tailored to each patient’s particular genetic profile.

  • Novel targeted therapies treat the root cause of CLL, resulting in lasting remissions and new treatment options for even the sickest patients.

  • New treatments go beyond symptom management to treat the underlying cause of PAH, allowing patients to maintain active lifestyles with reduced risk of serious heart events.

  • New discoveries in the underlying cause of HAE have led to breakthroughs in both preventative and acute treatment options for patients, including those targeting the root cause of the disease.

  • CF patients now have new treatment options allowing for better management of symptoms as well as a new option which allows many to target the underlying cause of their disease. If mortality rates continue to decline, patients may now hope to live into their 50s.

Thirty million Americans, or one in ten, and an estimated 350 million people worldwide are currently living with a rare disease, which is defined as a condition that affects fewer than 200,000 people. There are currently 7,000 known rare diseases and approximately 80 percent are caused by genetic abnormalities. However, despite incredible progress made against rare disease in recent years, only 5 percent have available treatment options.

For more information on rare diseases, view the “Medicines in Development for Rare Diseases” report. The full press release can be accessed here.