New therapies

ANTISENSE OLIGONUCLEOTIDE ELUFORSEN IS SAFE AND IMPROVES RESPIRATORY SYMPTOMS IN F508DEL CYSTIC FIBROSIS

Authors: Pavel Drevineka, Tacjana Presslerb, Marco Cipollic,d, Kris De Boecke, Carsten Schwarzf, Florilene Bouissetg,h, Marie Boffg, Noreen Henigi,j, Nicolas Paquette-Lamontagnei,k, Sonya Montgomeryi,l, Jaakko Perquini, Nigel Tomkinsoni, Wilhelmina den Hollanderi, and […]

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PILOT STUDY TO TEST INHALED NITRIC OXIDE IN CYSTIC FIBROSIS PATIENTS WITH REFRACTORY MYCOBACTERIUM ABSCESSUS LUNG INFECTION

Authors: Lea Bentura,b, Michal Gura,b, Moshe Ashkenazic,g, Galit Livnat-Levanond, Mark Mizrahie, Asher Tale, Abdi Ghaffarie, Yuval Geffenf, Micha Aviramg, Ori Efratic,h Affiliations: a Pediatric Pulmonary Institute and CF Center, Ruth […]

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ASSESSMENT OF ENDOTHELIAL FUNCTION IS REPRODUCIBLE IN PATIENTS WITH CYSTIC FIBROSIS

Authors: Cassandra C. Derella1,2, Nichole Lee2, Reva Crandall3, Marsha Blackburn2, Jacob Looney2, Amie Mangieri2, Paula Rodriguez-Miguelez4, Matthew A. Tucker2, Ryan A. Harris1,2,5 Affiliations: 1Department of Physiology, Augusta University, Augusta GA […]

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ESTABLISHMENT OF A F508DEL-CF PROMYELOCYTIC CELL LINE FOR CYSTIC FIBROSIS RESEARCH AND DRUG SCREENING

Authors: Scott Jennings, Hong Pong Ng and Guoshun Wang Affiliations: Department of Microbiology, Immunology and Parasitology, Louisiana State University Health Sciences Center, New Orleans, LA 70122, USA   What was […]

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CORRESPONDENCE BETWEEN SYMPTOMS AND PREFERENCE-BASED HEALTH STATUS MEASURES IN THE STOP STUDY

Authors: Laura S. Golda, Donald L. Patrickb, Ryan N. Hansenc, Valeria Beckettd, Christopher H. Gosse and Larry Kesslerb Affiliations: aDepartment of Radiology, University of Washington, Seattle, Washington, United States bDepartment […]

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ANTISENSE OLIGONUCLEOTIDE TARGETING OF MRNAS ENCODING ENAC SUBUNITS ALPHA, BETA, AND GAMMA IMPROVES CYSTIC FIBROSIS-LIKE DISEASE IN MICE

Authors: Chenguang Zhao, Jeff Crosby, Tinghong Lv, Dong Bai, Brett P. Monia, and Shuling Guo Affiliations: Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010 USA   What was your research […]

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HYPERBARIC OXYGEN TREATMENT INCREASES KILLING OF AGGREGATING PSEUDOMONAS AERUGINOSA ISOLATES FROM CYSTIC FIBROSIS PATIENTS

Authors: Møller, Signe Agnete1,2, Jensen, Peter Østrup1,2,3, Høiby, Niels1,2, Ciofu, Oana2, Kragh, Kasper Nørskov1,2, Bjarnsholt, Thomas1,2, Kolpen, Mette1,2 Affiliations: 1Department of Clinical Microbiology, Rigshospitalet, 2100 Copenhagen, Denmark 2Costerton Biofilm Center, […]

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SPX-101 IS STABLE IN AND RETAINS FUNCTION AFTER EXPOSURE TO CYSTIC FIBROSIS SPUTUM

Authors: Juliana I. Sesma, Bryant Wu, Timothy J. Stuhlmiller, David W. Scott Affiliations: Spyryx Biosciences, Durham, NC 27713   What was your research question? We wanted to understand the stability […]

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ELUFORSEN: A POTENTIAL NEW TREATMENT OPTION FOR PEOPLE WITH CYSTIC FIBROSIS (CF) DUE TO THE F508DEL GENE MUTATION?

Authors: Isabelle Sermet-Gaudelusa, John P. Clancyb, David P. Nicholsc, Jerry A. Nickd, Kris De Boecke, George M. Solomonf, Marcus A. Mallg,h,i, James Bolognesej, Florilene Bouissetk, Wilhelmina den Hollanderl, Nicolas Paquette-Lamontagnel,m, […]

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