CF Foundation President and CEO Robert J. Beall, Ph.D. testified today before the House of Representatives' Energy and Commerce Committee at a hearing entitled, “21st Century Cures: Incorporating the Patient Perspective.”
The U.S. Food and Drug Administration (FDA) has approved TOBI® Podhaler™ (tobramycin inhalation powder), a dry powder formulation of the antibiotic TOBI, to treat lung infections caused by the bacteria Pseudomonas aeruginosa (P. aeruginosa).
Legislation Would Increase Patient and Expert Participation in FDA Review of Rare Disease Treatments
The U.S. Food and Drug Administration (FDA) announced that it has approved Ultresa™ delayed-release capsules, a pancreatic enzyme replacement therapy. Ultresa (pancrelipase) is manufactured by Aptalis Pharma.
New Effort Will Search for Potential Therapies Targeting the Most Common Mutation of Cystic Fibrosis
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.