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Elexacaftor/tezacaftor/ivacaftor improves chronic rhinosinusitis detected by magnetic resonance imaging in children with cystic fibrosis on long-term therapy with lumacaftor/ivacaftor.
Wucherpfennig L, Becker JKZ, Wuennemann F, Eichinger M, Seitz A, Baumann I, Stahl M, Graeber SY, Zhao S, Chung J, Schenk JP, Alrajab A, Kauczor HU, Mall MA, Sommerburg O, Wielpütz MO. Wucherpfennig L, et al. Among authors: mall ma. J Cyst Fibros. 2024 Jan 12:S1569-1993(24)00004-3. doi: 10.1016/j.jcf.2024.01.004. Online ahead of print. J Cyst Fibros. 2024. PMID: 38218661 Free article.
Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis and at least one F508del allele: 144-week interim results from a 192-week open-label extension study.
Daines CL, Tullis E, Costa S, Linnemann RW, Mall MA, McKone EF, Polineni D, Quon BS, Ringshausen FC, Rowe SM, Selvadurai H, Taylor-Cousar JL, Withers NJ, Ahluwalia N, Moskowitz SM, Prieto-Centurion V, Tan YV, Tian S, Weinstock T, Xuan F, Zhang Y, Ramsey B, Griese M; VX17-445-105 Study Group. Daines CL, et al. Among authors: mall ma. Eur Respir J. 2023 Dec 7;62(6):2202029. doi: 10.1183/13993003.02029-2022. Print 2023 Dec. Eur Respir J. 2023. PMID: 37945033 Free PMC article. Clinical Trial.
BACKGROUND: In two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious and safe in patients with cystic fibrosis (CF) 12 years of age who have at least one F508del allele. The aim of this study is to assess long- …
BACKGROUND: In two pivotal phase 3 trials, up to 24 weeks of treatment with elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was efficacious a …
Effects of lumacaftor-ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2-11 years.
Berges J, Graeber SY, Hämmerling S, Yu Y, Krümpelmann A, Stahl M, Hirtz S, Scheuermann H, Mall MA, Sommerburg O. Berges J, et al. Among authors: mall ma. Front Pharmacol. 2023 May 30;14:1188051. doi: 10.3389/fphar.2023.1188051. eCollection 2023. Front Pharmacol. 2023. PMID: 37324488 Free PMC article.
Conclusion: Lumacaftor/ivacaftor partially restores F508del CFTR function in children with CF who are homozygous for F508del, aged 2-11 years, to a level of CFTR activity seen in patients with CFTR variants with residual function. These results are consistent with the partial sho …
Conclusion: Lumacaftor/ivacaftor partially restores F508del CFTR function in children with CF who are homozygous for F508del, aged 2-11 year …
Effects of elexacaftor/tezacaftor/ivacaftor therapy on mental health of patients with cystic fibrosis.
Piehler L, Thalemann R, Lehmann C, Thee S, Röhmel J, Syunyaeva Z, Stahl M, Mall MA, Graeber SY. Piehler L, et al. Among authors: mall ma. Front Pharmacol. 2023 Apr 21;14:1179208. doi: 10.3389/fphar.2023.1179208. eCollection 2023. Front Pharmacol. 2023. PMID: 37153809 Free PMC article.
Conclusion: Initiation of ETI improves symptoms of depression in adult patients with CF with at least one F508del allele. However, symptoms of anxiety do not change after short-term therapy with ETI....
Conclusion: Initiation of ETI improves symptoms of depression in adult patients with CF with at least one F508del allele. However, symptoms …
Long-term effects of lumacaftor/ivacaftor on paranasal sinus abnormalities in children with cystic fibrosis detected with magnetic resonance imaging.
Wucherpfennig L, Wuennemann F, Eichinger M, Seitz A, Baumann I, Stahl M, Graeber SY, Zhao S, Chung J, Schenk JP, Alrajab A, Kauczor HU, Mall MA, Sommerburg O, Wielpütz MO. Wucherpfennig L, et al. Among authors: mall ma. Front Pharmacol. 2023 Apr 10;14:1161891. doi: 10.3389/fphar.2023.1161891. eCollection 2023. Front Pharmacol. 2023. PMID: 37101549 Free PMC article.
Previous studies using magnetic resonance imaging (MRI) demonstrated early onset and progression of CRS from infancy to school age in patients with CF, and mid-term improvements of CRS in preschool and school-age children with CF treated with lumacaftor/ivacaftor for at le …
Previous studies using magnetic resonance imaging (MRI) demonstrated early onset and progression of CRS from infancy to school age in patien …
MDCT-based longitudinal automated airway and air trapping analysis in school-age children with mild cystic fibrosis lung disease.
Weinheimer O, Konietzke P, Wagner WL, Weber D, Newman B, Galbán CJ, Kauczor HU, Mall MA, Robinson TE, Wielpütz MO. Weinheimer O, et al. Among authors: mall ma. Front Pediatr. 2023 Feb 2;11:1068103. doi: 10.3389/fped.2023.1068103. eCollection 2023. Front Pediatr. 2023. PMID: 36816383 Free PMC article.
Further, BEI showed no cross-sectional, but another mixed model showed short-term longitudinal interdependencies with air trapping (P = 0.003). ...We could demonstrate cross-sectional, regional interdependencies between wall thickening and air trapping. Further, we show sh …
Further, BEI showed no cross-sectional, but another mixed model showed short-term longitudinal interdependencies with air trapping (P …
Magnetic resonance imaging-based scoring of the diseased lung in the preterm infant with bronchopulmonary dysplasia: UNiforme Scoring of the disEAsed Lung in BPD (UNSEAL BPD).
Förster K, Marchi H, Stöcklein S, Dietrich O, Ehrhardt H, Wielpütz MO, Flemmer AW, Schubert B, Mall MA, Ertl-Wagner B, Hilgendorff A. Förster K, et al. Among authors: mall ma. Am J Physiol Lung Cell Mol Physiol. 2023 Feb 1;324(2):L114-L122. doi: 10.1152/ajplung.00430.2021. Epub 2022 Nov 21. Am J Physiol Lung Cell Mol Physiol. 2023. PMID: 36410026 Free article.
Our novel MRI score enables the standardized assessment of disease-characteristic structural changes in the preterm lung exhibiting significant potential as a quantifiable endpoint in early intervention clinical trials and long-term disease monitoring....
Our novel MRI score enables the standardized assessment of disease-characteristic structural changes in the preterm lung exhibiting signific …
The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN.
Raidt J, Maitre B, Pennekamp P, Altenburg J, Anagnostopoulou P, Armengot M, Bloemsma LD, Boon M, Borrelli M, Brinkmann F, Carr SB, Carroll MP, Castillo-Corullón S, Coste A, Cutrera R, Dehlink E, Destouches DMS, Di Cicco ME, Dixon L, Emiralioglu N, Erdem Eralp E, Haarman EG, Hogg C, Karadag B, Kobbernagel HE, Lorent N, Mall MA, Marthin JK, Martinu V, Narayanan M, Ozcelik U, Peckham D, Pifferi M, Pohunek P, Polverino E, Range S, Ringshausen FC, Robson E, Roehmel J, Rovira-Amigo S, Santamaria F, Schlegtendal A, Szépfalusi Z, Tempels P, Thouvenin G, Ullmann N, Walker WT, Wetzke M, Yiallouros P, Omran H, Nielsen KG. Raidt J, et al. Among authors: mall ma. ERJ Open Res. 2022 Aug 15;8(3):00139-2022. doi: 10.1183/23120541.00139-2022. eCollection 2022 Jul. ERJ Open Res. 2022. PMID: 35983540 Free PMC article.
Macrophage PD-1 associates with neutrophilia and reduced bacterial killing in early cystic fibrosis airway disease.
Margaroli C, Horati H, Garratt LW, Giacalone VD, Schofield C, Dittrich AS, Rosenow T, Dobosh BS, Lim HS, Frey DL, Veltman M, Silva GL, Brown MR, Schultz C, Tiddens HAWM, Ranganathan S, Chandler JD, Qiu P, Peng L, Scholte BJ, Mall MA, Kicic A, Guglani L, Stick SM, Janssens HM, Tirouvanziam R. Margaroli C, et al. Among authors: mall ma. J Cyst Fibros. 2022 Nov;21(6):967-976. doi: 10.1016/j.jcf.2022.06.001. Epub 2022 Jun 19. J Cyst Fibros. 2022. PMID: 35732550 Free article.
In cystic fibrosis (CF), neutrophils are recruited to the airways shortly after birth, and actively exocytose damaging enzymes prior to chronic infection, suggesting a potential defect in macrophage immunomodulatory function. ...In an in vitro model mimicking leukocyte rec …
In cystic fibrosis (CF), neutrophils are recruited to the airways shortly after birth, and actively exocytose damaging enzymes prior …
Changes in Microbiome Dominance Are Associated With Declining Lung Function and Fluctuating Inflammation in People With Cystic Fibrosis.
Frey DL, Bridson C, Dittrich S, Graeber SY, Stahl M, Wege S, Herth F, Sommerburg O, Schultz C, Dalpke A, Mall MA, Boutin S. Frey DL, et al. Among authors: mall ma. Front Microbiol. 2022 May 13;13:885822. doi: 10.3389/fmicb.2022.885822. eCollection 2022. Front Microbiol. 2022. PMID: 35633718 Free PMC article.
However, longitudinal studies are needed to decipher which factors contribute to the long-term evolution of these key features of CF. We therefore evaluated the relationship between fluctuation in microbiome and inflammatory parameters in a longitudinal study including a s …
However, longitudinal studies are needed to decipher which factors contribute to the long-term evolution of these key features of CF. …
17 results