FDA Expands Orphan Drug Indication Based on Lab Tests
Janet Woodcock: “Many rare cystic fibrosis mutations have such small patient populations that clinical trial studies are not feasible"
The Mauli Ola Foundation (MOF) are a team surfers who help cystic fibrosis patients breathe better. And it is based on solid scientific evidence.
Born without a lower jaw, Isaiah can’t breathe through his mouth and he has never spoken a word. In spite of it, the 17-year-old released his first rap song.
BPompe disease is a rare lysosomal storage disorder that impacts muscle strength. Weak muscles of the airways and tongue can affect patients' ability to talk, breath, and swallow.
Data from two Phase 3 cystic fibrosis study indicated Vertex’s new combination therapy (tezacaftor / ivacaftor) will be a winner.
Today, the company announced their pivotal cystic fibrosis trial with ataluran failed to meet its primary endpoint.
The App was developed by Misha Rosenbach, MD and Daniel O’Connor, a fourth-year medical student at University of Pennsylvania.
CTP-656, using a novel application of deuterium chemistry, is a modified version of Kalydeco
Cystic fibrosis patients often develop EPI as a result of the mucus build up that occurs in the pancreas that leads to decreased exocrine pancreatic enzymes being released.
At the Child Neurology Society Annual Meeting (CNS 2016) in Vancouver, we talked with James Meyer, Senior Director of Medical Affairs at Marathon Pharmaceuticals about their poster presentation
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