Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...
A nanobody that stabilizes mutant CFTR from inside the cell, especially when combined with existing drugs, restores near-normal function in cystic fibrosis cells.
Children ages two to five who have the most common form of cystic fibrosis (CF), caused by two copies of the F508 gene mutation, have not had any modulator treatments available to them until recently.
BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX ...
Over the course of two decades spent developing treatments for the genetic lung disease cystic fibrosis, biologist Fredrick Van Goor has had hundreds of conversations with patients. But he remembers ...
The drug is a small molecule with a novel mechanism of action, say David Bedwell, Ph.D., and Steven Rowe, M.D., MSPH, co-senior authors. Bedwell is professor and chair of the University of Alabama at ...
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