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An evaluation of an open access iPSC training course: "How to model interstitial lung disease using patient-derived iPSCs".
Schweikert A, Kenny S, Oglesby I, Glasgow A, de Santi C, Gensch I, Lachmann N, Desroziers T, Fletcher C, Snijders D, Nathan N, Hurley K; COST Open-ILD Group Management Committee. Schweikert A, et al. Stem Cell Res Ther. 2023 Dec 20;14(1):377. doi: 10.1186/s13287-023-03598-9. Stem Cell Res Ther. 2023. PMID: 38124115 Free PMC article.
Overall, our findings demonstrate the feasibility of running hybrid Hands-on and Observer teaching events and underscore the importance of this type of training programme to appeal to a broad spectrum of interested clinicians and researchers particularly in rare disease. The long …
Overall, our findings demonstrate the feasibility of running hybrid Hands-on and Observer teaching events and underscore the importance of t …
Towards personalized therapies for genetic disorders of surfactant dysfunction.
Peers de Nieuwburgh M, Wambach JA, Griese M, Danhaive O. Peers de Nieuwburgh M, et al. Among authors: griese m. Semin Fetal Neonatal Med. 2023 Dec;28(6):101500. doi: 10.1016/j.siny.2023.101500. Epub 2023 Nov 22. Semin Fetal Neonatal Med. 2023. PMID: 38036307 Review.
Genetic disorders of surfactant dysfunction are a rare cause of chronic, progressive or refractory respiratory failure in term and preterm infants. This review explores genetic mechanisms underpinning surfactant dysfunction, highlighting specific surfactant-associated gene …
Genetic disorders of surfactant dysfunction are a rare cause of chronic, progressive or refractory respiratory failure in term and pr …
Inhaled recombinant GM-CSF reduces the need for whole lung lavage and improves gas exchange in autoimmune pulmonary alveolar proteinosis patients.
Campo I, Carey BC, Paracchini E, Kadija Z, De Silvestri A, Rodi G, De Amici M, Torre C, Zorzetto M, Griese M, Meloni F, Corsico AG, Trapnell BC, Mariani F. Campo I, et al. Among authors: griese m. Eur Respir J. 2024 Jan 4;63(1):2301233. doi: 10.1183/13993003.01233-2023. Print 2024 Jan. Eur Respir J. 2024. PMID: 37973175 Free PMC article. Clinical Trial.
No serious adverse events were reported. CONCLUSIONS: This long-term, prospective, randomised trial demonstrated inhaled sargramostim following WLL reduced the requirement for WLL, improved lung function and was safe in aPAP patients. ...
No serious adverse events were reported. CONCLUSIONS: This long-term, prospective, randomised trial demonstrated inhaled sargramostim …
The Human Phenotype Ontology in 2024: phenotypes around the world.
Gargano MA, Matentzoglu N, Coleman B, Addo-Lartey EB, Anagnostopoulos AV, Anderton J, Avillach P, Bagley AM, Bakštein E, Balhoff JP, Baynam G, Bello SM, Berk M, Bertram H, Bishop S, Blau H, Bodenstein DF, Botas P, Boztug K, Čady J, Callahan TJ, Cameron R, Carbon SJ, Castellanos F, Caufield JH, Chan LE, Chute CG, Cruz-Rojo J, Dahan-Oliel N, Davids JR, de Dieuleveult M, de Souza V, de Vries BBA, de Vries E, DePaulo JR, Derfalvi B, Dhombres F, Diaz-Byrd C, Dingemans AJM, Donadille B, Duyzend M, Elfeky R, Essaid S, Fabrizzi C, Fico G, Firth HV, Freudenberg-Hua Y, Fullerton JM, Gabriel DL, Gilmour K, Giordano J, Goes FS, Moses RG, Green I, Griese M, Groza T, Gu W, Guthrie J, Gyori B, Hamosh A, Hanauer M, Hanušová K, He YO, Hegde H, Helbig I, Holasová K, Hoyt CT, Huang S, Hurwitz E, Jacobsen JOB, Jiang X, Joseph L, Keramatian K, King B, Knoflach K, Koolen DA, Kraus ML, Kroll C, Kusters M, Ladewig MS, Lagorce D, Lai MC, Lapunzina P, Laraway B, Lewis-Smith D, Li X, Lucano C, Majd M, Marazita ML, Martinez-Glez V, McHenry TH, McInnis MG, McMurry JA, Mihulová M, Millett CE, Mitchell PB, Moslerová V, Narutomi K, Nematollahi S, Nevado J, Nierenberg AA, Čajbiková NN, Nurnberger JI Jr, Og… See abstract for full author list ➔ Gargano MA, et al. Among authors: griese m. Nucleic Acids Res. 2024 Jan 5;52(D1):D1333-D1346. doi: 10.1093/nar/gkad1005. Nucleic Acids Res. 2024. PMID: 37953324 Free PMC article.
In recent years, groups around the world have developed translations of the HPO from English to other languages, and the HPO browser has been internationalized, allowing users to view HPO term labels and in many cases synonyms and definitions in ten languages in addition t …
In recent years, groups around the world have developed translations of the HPO from English to other languages, and the HPO browser has bee …
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: griese m. 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 …
Severe Neonatal Interstitial Lung Disease Caused by a Rare Surfactant Protein C Mutation.
Terpe F, Schwerk N, Griese M, Laenger Florian P, Ballmann M, Chao CM, Ehler J. Terpe F, et al. Among authors: griese m. Pediatrics. 2023 Jun 1;151(6):e2022060038. doi: 10.1542/peds.2022-060038. Pediatrics. 2023. PMID: 37232099
Childhood interstitial lung disease (chILD) is a collective term for a group of rare lung disorders of heterogeneous origin. ...Clinical signs of tachypnea and hypoxemia are nonspecific and usually caused by common conditions like lower respiratory tract infections. We rep …
Childhood interstitial lung disease (chILD) is a collective term for a group of rare lung disorders of heterogeneous origin. ...Clini …
Evaluation of Autoantibody Binding to Cardiac Tissue in Multisystem Inflammatory Syndrome in Children and COVID-19 Vaccination-Induced Myocarditis.
Patel H, Sintou A, Chowdhury RA, Rothery S, Iacob AO, Prasad S, Rainer PP, Martinón-Torres F, Sancho-Shimizu V, Shimizu C, Dummer K, Tremoulet AH, Burns JC, Sattler S, Levin M; DIAMONDS consortium. Patel H, et al. JAMA Netw Open. 2023 May 1;6(5):e2314291. doi: 10.1001/jamanetworkopen.2023.14291. JAMA Netw Open. 2023. PMID: 37200028 Free PMC article.
Diffuse alveolar haemorrhage in children: an international multicentre study.
Ring AM, Schwerk N, Kiper N, Aslan AT, Aurora P, Ayats R, Azevedo I, Bandeira T, Carlens J, Castillo-Corullon S, Cobanoglu N, Elnazir B, Emiralioğlu N, Eyuboglu TS, Fayon M, Gursoy TR, Hogg C, Kötz K, Karadag B, Látalová V, Krenke K, Lange J, Manali ED, Osona B, Papiris S, Proesmann M, Reix P, Roditis L, Rubak S, Rumman N, Snijders D, Stehling F, Weiss L, Yalcın E, Zirek F, Bush A, Clement A, Griese M, Buchvald FF, Nathan N, Nielsen KG. Ring AM, et al. Among authors: griese m. ERJ Open Res. 2023 Apr 24;9(2):00733-2022. doi: 10.1183/23120541.00733-2022. eCollection 2023 Mar. ERJ Open Res. 2023. PMID: 37101741 Free PMC article.
The most frequent medical treatment was systemic corticosteroids (93%), hydroxychloroquine (35%) and azathioprine (27%). Overall mortality was 13%. Long-term data demonstrated persistent abnormal radiology and a limited improvement in lung function. ...This large internati …
The most frequent medical treatment was systemic corticosteroids (93%), hydroxychloroquine (35%) and azathioprine (27%). Overall mortality w …
ABCA3-related interstitial lung disease beyond infancy.
Li Y, Seidl E, Knoflach K, Gothe F, Forstner ME, Michel K, Pawlita I, Gesenhues F, Sattler F, Yang X, Kroener C, Reu-Hofer S, Ley-Zaporozhan J, Kammer B, Krüger-Stollfuß I, Dinkel J, Carlens J, Wetzke M, Moreno-Galdó A, Torrent-Vernetta A, Lange J, Krenke K, Rumman N, Mayell S, Sismanlar T, Aslan A, Regamey N, Proesmans M, Stehling F, Naehrlich L, Ayse K, Becker S, Koerner-Rettberg C, Plattner E, Manali ED, Papiris SA, Campo I, Kappler M, Schwerk N, Griese M. Li Y, et al. Among authors: griese m. Thorax. 2023 Jun;78(6):587-595. doi: 10.1136/thorax-2022-219434. Epub 2023 Feb 20. Thorax. 2023. PMID: 36808083 Free PMC article.
METHOD: Over a 21-year period, patients diagnosed as chILD due to ABCA3 deficiency were identified from the Kids Lung Register database. 44 patients survived beyond the first year of life and their long-term clinical course, oxygen supplementation and pulmonary function we …
METHOD: Over a 21-year period, patients diagnosed as chILD due to ABCA3 deficiency were identified from the Kids Lung Register database. 44 …
European Respiratory Society statement for defining respiratory exacerbations in children and adolescents with bronchiectasis for clinical trials.
Chang AB, Zacharasiewicz A, Goyal V, Boyd J, Alexopoulou E, Aliberti S, Bell L, Bush A, Claydon A, Constant C, Fortescue R, Hill AT, Karadag B, Powell Z, Wilson C, Grimwood K, Kantar A; other members of Child-BEAR-Net; Chalmers J, Collaro A, Douros K, Griese M, Grigg J, Hector A, Mazulov O, Midulla F, Möller A, Proesmans M, Yerkovich S. Chang AB, et al. Among authors: griese m. Eur Respir J. 2022 Nov 3;60(5):2200300. doi: 10.1183/13993003.00300-2022. Print 2022 Nov. Eur Respir J. 2022. PMID: 35728974
Respiratory exacerbations are associated with an impaired quality of life, poorer long-term clinical outcomes, and substantial costs to the family and health systems. ...
Respiratory exacerbations are associated with an impaired quality of life, poorer long-term clinical outcomes, and substantial costs …
57 results