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1.

A randomised trial of oral prednisone for cystic fibrosis pulmonary exacerbation treatment.

Waters, Valerie; Shaw, Michelle; Perrem, Lucy; Quon, Bradley S; Tullis, Elizabeth; Solomon, Melinda; Rayment, Jonathan H; Lavoie, Annick; Tse, Sze Man; Daigneault, Patrick; Bilodeau, Lara; Price, April; Nicholson, Michael; Chin, Melanie; Parkins, Michael; McKinney, Martha L; Tam, Julian S; Stanojevic, Sanja; Grasemann, Hartmut; Ratjen, Felix.

Eur Respir J ; 63(6)2024 Jun.

Artículo en Inglés | MEDLINE | ID: mdl-38697648

2.

Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele.

Goralski, Jennifer L; Hoppe, Jordana E; Mall, Marcus A; McColley, Susanna A; McKone, Edward; Ramsey, Bonnie; Rayment, Jonathan H; Robinson, Phil; Stehling, Florian; Taylor-Cousar, Jennifer L; Tullis, Elizabeth; Ahluwalia, Neil; Chin, Anna; Chu, Chenghao; Lu, Mengdi; Niu, Tao; Weinstock, Tanya; Ratjen, Felix; Rosenfeld, Margaret.

Am J Respir Crit Care Med ; 208(1): 59-67, 2023 07 01.

Artículo en Inglés | MEDLINE | ID: mdl-36921081

3.

Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged â©¾6 Years with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Clinical Trial.

Wainwright, Claire; McColley, Susanna A; McNally, Paul; Powers, Michael; Ratjen, Felix; Rayment, Jonathan H; Retsch-Bogart, George; Roesch, Erica; Ahluwalia, Neil; Chin, Anna; Chu, Chenghao; Lu, Mengdi; Menon, Prema; Waltz, David; Weinstock, Tanya; Zelazoski, Laura; Davies, Jane C.

Am J Respir Crit Care Med ; 208(1): 68-78, 2023 07 01.

Artículo en Inglés | MEDLINE | ID: mdl-37154609

4.

Brain AVM compactness score in children with hereditary hemorrhagic telangiectasia.

Beslow, Lauren A; Vossough, Arastoo; Kim, Helen; Nelson, Jeffrey; Lawton, Michael T; Pollak, Jeffrey; Lin, Doris D M; Ratjen, Felix; Hammill, Adrienne M; Hetts, Steven W; Gossage, James R; Whitehead, Kevin J; Faughnan, Marie E; Krings, Timo.

Childs Nerv Syst ; 2024 Mar 22.

Artículo en Inglés | MEDLINE | ID: mdl-38517485

5.

Evaluation of clinically relevant changes in the lung clearance index in children with cystic fibrosis and healthy controls.

Perrem, Lucy; Stanojevic, Sanja; Solomon, Melinda; Grasemann, Hartmut; Sweezey, Neil; Waters, Valerie; Sanders, Don B; Davis, Stephanie D; Ratjen, Felix.

Thorax ; 78(4): 362-367, 2023 04.

Artículo en Inglés | MEDLINE | ID: mdl-35428702

6.

A 3D stack-of-spirals approach for rapid hyperpolarized ¹²⁹ Xe ventilation mapping in pediatric cystic fibrosis lung disease.

Zanette, Brandon; Munidasa, Samal; Friedlander, Yonni; Ratjen, Felix; Santyr, Giles.

Magn Reson Med ; 89(3): 1083-1091, 2023 03.

Artículo en Inglés | MEDLINE | ID: mdl-36433705

7.

Inter- and intravisit repeatability of free-breathing MRI in pediatric cystic fibrosis lung disease.

Munidasa, Samal; Zanette, Brandon; Couch, Marcus; Grimm, Robert; Seethamraju, Ravi; Dumas, Marie-Pier; Wee, Wallace; Au, Jacky; Braganza, Sharon; Li, Daniel; Woods, Jason; Ratjen, Felix; Santyr, Giles.

Magn Reson Med ; 89(5): 2048-2061, 2023 05.

Artículo en Inglés | MEDLINE | ID: mdl-36576212

8.

Longitudinal Assessment of Curaçao Criteria in Children with Hereditary Hemorrhagic Telangiectasia.

Pollak, Mordechai; Gatt, Dvir; Shaw, Michelle; Hewko, Sheryl L; Lamanna, Anthony; Santos, Sara; Ratjen, Felix.

J Pediatr ; 263: 113665, 2023 Dec.

Artículo en Inglés | MEDLINE | ID: mdl-37572862

9.

Intra- and Inter-visit Repeatability of ¹²⁹ Xenon Multiple-Breath Washout MRI in Children With Stable Cystic Fibrosis Lung Disease.

Alam, Faiyza S; Zanette, Brandon; Munidasa, Samal; Braganza, Sharon; Li, Daniel; Woods, Jason C; Ratjen, Felix; Santyr, Giles.

J Magn Reson Imaging ; 58(3): 936-948, 2023 09.

Artículo en Inglés | MEDLINE | ID: mdl-36786650

10.

Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial.

Nichols, David P; Paynter, Alex C; Heltshe, Sonya L; Donaldson, Scott H; Frederick, Carla A; Freedman, Steven D; Gelfond, Daniel; Hoffman, Lucas R; Kelly, Andrea; Narkewicz, Michael R; Pittman, Jessica E; Ratjen, Felix; Rosenfeld, Margaret; Sagel, Scott D; Schwarzenberg, Sarah Jane; Singh, Pradeep K; Solomon, George M; Stalvey, Michael S; Clancy, John P; Kirby, Shannon; Van Dalfsen, Jill M; Kloster, Margaret H; Rowe, Steven M.

Am J Respir Crit Care Med ; 205(5): 529-539, 2022 03 01.

Artículo en Inglés | MEDLINE | ID: mdl-34784492

11.

Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508del and a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study.

Mall, Marcus A; Brugha, Rossa; Gartner, Silvia; Legg, Julian; Moeller, Alexander; Mondejar-Lopez, Pedro; Prais, Dario; Pressler, Tacjana; Ratjen, Felix; Reix, Philippe; Robinson, Paul D; Selvadurai, Hiran; Stehling, Florian; Ahluwalia, Neil; Arteaga-Solis, Emilio; Bruinsma, Bote G; Jennings, Mark; Moskowitz, Samuel M; Noel, Sabrina; Tian, Simon; Weinstock, Tanya G; Wu, Pan; Wainwright, Claire E; Davies, Jane C.

Am J Respir Crit Care Med ; 206(11): 1361-1369, 2022 12 01.

Artículo en Inglés | MEDLINE | ID: mdl-35816621

12.

Lower Airway Nitrogen Oxide Levels in Children with Primary Ciliary Dyskinesia Is Linked to Neutrophilic Inflammation.

Grasemann, Hartmut; McDonald, Nancy; Yuan, Xi Zhou; Dell, Sharon; Waters, Valerie; Ratjen, Felix.

J Pediatr ; 244: 230-233, 2022 05.

Artículo en Inglés | MEDLINE | ID: mdl-35120987

13.

Lung Clearance Index to Track Acute Respiratory Events in School-Age Children with Cystic Fibrosis.

Perrem, Lucy; Stanojevic, Sanja; Shaw, Michelle; Jensen, Renee; McDonald, Nancy; Isaac, Sarah M; Davis, Miriam; Clem, Charles; Guido, Julia; Jara, Sylvia; France, Lisa; Solomon, Melinda; Grasemann, Hartmut; Waters, Valerie; Sweezey, Neil; Sanders, Don B; Davis, Stephanie D; Ratjen, Felix.

Am J Respir Crit Care Med ; 203(8): 977-986, 2021 04 15.

Artículo en Inglés | MEDLINE | ID: mdl-33030967

14.

Cystic Fibrosis.

Grasemann, Hartmut; Ratjen, Felix.

N Engl J Med ; 389(18): 1693-1707, 2023 Nov 02.

Artículo en Inglés | MEDLINE | ID: mdl-37913507

15.

Time to get serious about the detection and monitoring of early lung disease in cystic fibrosis.

Bayfield, Katie J; Douglas, Tonia A; Rosenow, Tim; Davies, Jane C; Elborn, Stuart J; Mall, Marcus; Paproki, Anthony; Ratjen, Felix; Sly, Peter D; Smyth, Alan R; Stick, Stephen; Wainwright, Claire E; Robinson, Paul D.

Thorax ; 76(12): 1255-1265, 2021 12.

Artículo en Inglés | MEDLINE | ID: mdl-33927017

16.

Unsupervised phenotypic clustering for determining clinical status in children with cystic fibrosis.

Filipow, Nicole; Davies, Gwyneth; Main, Eleanor; Sebire, Neil J; Wallis, Colin; Ratjen, Felix; Stanojevic, Sanja.

Eur Respir J ; 58(2)2021 08.

Artículo en Inglés | MEDLINE | ID: mdl-33446607

17.

Determinants of lung disease progression measured by lung clearance index in children with cystic fibrosis.

Stanojevic, Sanja; Davis, Stephanie D; Perrem, Lucy; Shaw, Michelle; Retsch-Bogart, George; Davis, Miriam; Jensen, Renee; Clem, Charles C; Isaac, Sarah M; Guido, Julia; Jara, Sylvia; France, Lisa; McDonald, Nancy; Solomon, Melinda; Sweezey, Neil; Grasemann, Hartmut; Waters, Valerie; Sanders, D B; Ratjen, Felix A.

Eur Respir J ; 58(1)2021 07.

Artículo en Inglés | MEDLINE | ID: mdl-33542049

18.

Second International Guidelines for the Diagnosis and Management of Hereditary Hemorrhagic Telangiectasia.

Faughnan, Marie E; Mager, Johannes J; Hetts, Steven W; Palda, Valerie A; Lang-Robertson, Kelly; Buscarini, Elisabetta; Deslandres, Erik; Kasthuri, Raj S; Lausman, Andrea; Poetker, David; Ratjen, Felix; Chesnutt, Mark S; Clancy, Marianne; Whitehead, Kevin J; Al-Samkari, Hanny; Chakinala, Murali; Conrad, Miles; Cortes, Daniel; Crocione, Claudia; Darling, Jama; de Gussem, Els; Derksen, Carol; Dupuis-Girod, Sophie; Foy, Patrick; Geisthoff, Urban; Gossage, James R; Hammill, Adrienne; Heimdal, Ketil; Henderson, Katharine; Iyer, Vivek N; Kjeldsen, Anette D; Komiyama, Masaki; Korenblatt, Kevin; McDonald, Jamie; McMahon, Jack; McWilliams, Justin; Meek, Mary E; Mei-Zahav, Meir; Olitsky, Scott; Palmer, Sara; Pantalone, Rose; Piccirillo, Jay F; Plahn, Beth; Porteous, Mary E M; Post, Marco C; Radovanovic, Ivan; Rochon, Paul J; Rodriguez-Lopez, Josanna; Sabba, Carlo; Serra, Marcelo.

Ann Intern Med ; 173(12): 989-1001, 2020 12 15.

Artículo en Inglés | MEDLINE | ID: mdl-32894695

19.

Primary care providers' role in newborn screening result notification for cystic fibrosis.

Hayeems, Robin Z; Miller, Fiona A; Barg, Carolyn J; Bombard, Yvonne; Chakraborty, Pranesh; Potter, Beth K; Patton, Sarah; Bytautas, Jessica Peace; Tam, Karen; Taylor, Louise; Kerr, Elizabeth; Davies, Christine; Milburn, Jennifer; Ratjen, Felix; Guttmann, Astrid; Carroll, June C.

Can Fam Physician ; 67(6): 439-448, 2021 06.

Artículo en Inglés | MEDLINE | ID: mdl-34127469

20.

Newborn screening for cystic fibrosis: Role of primary care providers in caring for infants with positive screening results.

Carroll, June C; Hayeems, Robin Z; Miller, Fiona A; Barg, Carolyn J; Bombard, Yvonne; Chakraborty, Pranesh; Potter, Beth K; Bytautas, Jessica Peace; Tam, Karen; Taylor, Louise; Kerr, Elizabeth; Davies, Christine; Milburn, Jennifer; Ratjen, Felix; Guttmann, Astrid.

Can Fam Physician ; 67(6): e144-e152, 2021 06.

Artículo en Inglés | MEDLINE | ID: mdl-34127476

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