The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
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The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
A new law that allows patients with rare diseases to participate in clinical trials without losing eligibility for public health care benefits went into effect yesterday. The bill, known as the “Improving Access to Clinical Trials Act” (IACT), was championed by the Cystic Fibrosis Foundation and signed into law in October 2010.
The Cystic Fibrosis Foundation's successful business model was at the center of a congressional briefing in Washington, D.C., today, which focused on strategies for jump-starting drug development for rare diseases.