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Making Treatment Less Rare for Rare Disease Patients

Waiting is a fact of life for patients with rare diseases such as cystic fibrosis.

Many of these men and women, whose disease causes wheezing and frequent lung infections, live waiting for new and effective medications.  They have to delay activities due to symptoms that make breathing a struggle.  They wait for treatment, fighting through health plan barriers such as prior authorization or awaiting the decision of opaque bureaucracies such as Medicaid pharmacy and therapeutics committees.