Dr. Frank Accurso, director of cystic fibrosis clinical research at Children's Hospital Colorado and University of Colorado Denver, participated in a congressional roundtable discussion at National Jewish Health in Denver, Colorado.
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Researchers' catalog of airway cell types could reveal targets for future genetic therapies
The Cystic Fibrosis Foundation celebrates the FDA's approval today of the lumacaftor/ivacaftor combination drug (Orkambi™) as an important advance for the cystic fibrosis community. Orkambi targets the underlying cause of the disease in people with two copies of the most common CF gene mutation.
Today, the Cystic Fibrosis Foundation announced that it will invest up to $8.4 million in SpliSense's Series B funding round to develop an antisense oligonucleotide therapy for people with cystic fibrosis who have splicing mutations and potentially other rare mutations.
Foundation to fund up to $110 million for collaboration to accelerate the development of genetic therapies for CF
The Cystic Fibrosis Foundation participated in a Congressional briefing hosted by Friends of Cancer Research on July 24 to educate members of the U.S. Senate about new processes developed by the FDA to speed the approval of breakthrough therapies.
Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.
Results show that people on Trikafta® enrolled in the six-week study were able to safely stop taking one of the two common CF medications without negatively affecting their health.