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Home telemonitoring makes early hospital discharge of COVID-19 patients possible

Journal of the American Medical Informatics Association : JAMIA, 2020-11, Vol.27 (11), p.1825-1827

2020

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Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

Science translational medicine, 2016-06, Vol.8 (344), p.344ra84

2016

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Long-term abatacept treatment for 48 weeks in patients with primary Sjögren's syndrome: The open-label extension phase of the ASAP-III trial

Seminars in arthritis and rheumatism, 2022-04, Vol.53, p.151955-151955, Article 151955

2022

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Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

Cell reports (Cambridge), 2019-02, Vol.26 (7), p.1701-1708.e3

2019

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Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

The Lancet (British edition), 2019-11, Vol.394 (10212), p.1940-1948

2019

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Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

The New England journal of medicine, 2019-11, Vol.381 (19), p.1809-1819

2019

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Targeted proteomics and metabolomics for biomarker discovery in abdominal aortic aneurysm and post-EVAR sac volume

Clinica chimica acta, 2024-02, Vol.554, p.117786-117786, Article 117786

2024

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Targeted plasma multi-omics propose glutathione, glycine and serine as biomarkers for abdominal aortic aneurysm growth on serial CT scanning

Atherosclerosis, 2024-11, Vol.398, p.118620, Article 118620

2024

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The clinical and pathological phenotype of C9ORF72 hexanucleotide repeat expansions

Brain (London, England : 1878), 2012-03, Vol.135 (Pt 3), p.723-735

2012

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Circulating biomarkers of cardiovascular disease are related to aneurysm volume in abdominal aortic aneurysm

Vascular medicine (London, England), 2023-10, Vol.28 (5), p.433-442

2023

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Strength Properties of Grout for Strata Reinforcement

Procedia engineering, 2017, Vol.191, p.1178-1184

2017

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Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation

Journal of cystic fibrosis, 2020-11, Vol.19 (6), p.955-961

2020

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Distress in partners of individuals diagnosed with or at high risk of developing tumors due to rare hereditary cancer syndromes

Psycho-oncology (Chichester, England), 2011-06, Vol.20 (6), p.631-638

2011

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Inflammation and TGF-β Signaling Differ between Abdominal Aneurysms and Occlusive Disease

Journal of cardiovascular development and disease, 2019-11, Vol.6 (4), p.38

2019

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Molecular phenotyping and functional assessment of smooth muscle-like cells with pathogenic variants in aneurysm genes ACTA2, MYH11, SMAD3 and FBN1

Human molecular genetics, 2021-11, Vol.30 (23), p.2286-2299

2021

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Update of the pompe disease mutation database with 60 novel GAA sequence variants and additional studies on the functional effect of 34 previously reported variants

Human mutation, 2012-08, Vol.33 (8), p.1161-1165

2012

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S65 Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis heterozygous for F508del and a minimal function mutation (F/MF): results from a Phase 3 clinical study

Thorax, 2021-02, Vol.76 (Suppl 1), p.A40-A41

2021

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S66 Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis homozygous for F508del (F/F): results from a Phase 3 clinical study

Thorax, 2021-02, Vol.76 (Suppl 1), p.A41-A42

2021

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Postnatal Outcome of Fetal Left Ventricular Hypertrabeculation/Noncompaction

Pediatric cardiology, 2016-06, Vol.37 (5), p.919-924

2016

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Individual and Group Response of Treatment with Ivacaftor on Airway and Gut Microbiota in People with CF and a S1251N Mutation

Journal of personalized medicine, 2021-04, Vol.11 (5), p.350

2021

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β 2 -Adrenergic receptor agonists activate CFTR in intestinal organoids and subjects with cystic fibrosis

The European respiratory journal, 2016-09, Vol.48 (3), p.768-779

2016

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P221 Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis heterozygous for F508del and a minimal function mutation: results from a Phase 3 clinical study

Journal of cystic fibrosis, 2020-06, Vol.19, p.S118-S119

2020

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WS19.6 Impact of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) triple combination therapy on health-related quality of life (HRQoL) in people with cystic fibrosis (pwCF) homozygous for F508del (F/F): results from a Phase 3 clinical study

Journal of cystic fibrosis, 2020-06, Vol.19, p.S32-S32

2020

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P207 Start of lumacaftor/ivacaftor in adult cystic fibrosis patients with homozygous F508del results in temporal and moderate changes in lung microbiome and metabolome

Journal of cystic fibrosis, 2020-06, Vol.19, p.S115-S115

2020

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Home telemonitoring makes early hospital discharge of COVID-19 patients possible

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

Long-term abatacept treatment for 48 weeks in patients with primary Sjögren's syndrome: The open-label extension phase of the ASAP-III trial

Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

Targeted proteomics and metabolomics for biomarker discovery in abdominal aortic aneurysm and post-EVAR sac volume

Targeted plasma multi-omics propose glutathione, glycine and serine as biomarkers for abdominal aortic aneurysm growth on serial CT scanning

The clinical and pathological phenotype of C9ORF72 hexanucleotide repeat expansions

Circulating biomarkers of cardiovascular disease are related to aneurysm volume in abdominal aortic aneurysm

Strength Properties of Grout for Strata Reinforcement

Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation

Distress in partners of individuals diagnosed with or at high risk of developing tumors due to rare hereditary cancer syndromes

Inflammation and TGF-β Signaling Differ between Abdominal Aneurysms and Occlusive Disease

Molecular phenotyping and functional assessment of smooth muscle-like cells with pathogenic variants in aneurysm genes ACTA2, MYH11, SMAD3 and FBN1

Update of the pompe disease mutation database with 60 novel GAA sequence variants and additional studies on the functional effect of 34 previously reported variants

S65 Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis heterozygous for F508del and a minimal function mutation (F/MF): results from a Phase 3 clinical study

S66 Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis homozygous for F508del (F/F): results from a Phase 3 clinical study

Manifest disease, risk factors for sudden cardiac death, and cardiac events in a large nationwide cohort of predictively tested hypertrophic cardiomyopathy mutation carriers: determining the best cardiological screening strategy

Postnatal Outcome of Fetal Left Ventricular Hypertrabeculation/Noncompaction

Individual and Group Response of Treatment with Ivacaftor on Airway and Gut Microbiota in People with CF and a S1251N Mutation

β 2 -Adrenergic receptor agonists activate CFTR in intestinal organoids and subjects with cystic fibrosis

P221 Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis heterozygous for F508del and a minimal function mutation: results from a Phase 3 clinical study

WS19.6 Impact of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) triple combination therapy on health-related quality of life (HRQoL) in people with cystic fibrosis (pwCF) homozygous for F508del (F/F): results from a Phase 3 clinical study

P207 Start of lumacaftor/ivacaftor in adult cystic fibrosis patients with homozygous F508del results in temporal and moderate changes in lung microbiome and metabolome

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