A multicenter Italian study and an accompanying commentary provide real-world evidence that early lumacaftor/ivacaftor ...
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A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...
UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new interim data from the ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) 2 to 5 years old open-label extension study, highlighting ...
Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...
BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that data on TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor ...
MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, ...
Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...
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