نتائج البحث

Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype.

• Authors : Terlizzi V; Cystic Fibrosis Regional Reference Center,, Department of Paediatric Medicine, Anna Meyer Children's University, Florence, Italy.; Amato F

Subjects: Genotype*; Aminophenols/Aminophenols/Aminophenols/*therapeutic use ; Aminopyridines/Aminopyridines/Aminopyridines/*therapeutic use

• Source: Molecular genetics & genomic medicine [Mol Genet Genomic Med] 2021 Apr; Vol. 9 (4), pp. e1656. Date of Electronic Publication: 2021 Mar 13.Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 101603758 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene.

• Authors : Venturini A; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.; Borrelli A

Subjects: Codon, Nonsense*; Aminopyridines/Aminopyridines/Aminopyridines/*pharmacology ; Benzodioxoles/Benzodioxoles/Benzodioxoles/*pharmacology

• Source: International journal of molecular sciences [Int J Mol Sci] 2021 Nov 04; Vol. 22 (21). Date of Electronic Publication: 2021 Nov 04.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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GM1 as Adjuvant of Innovative Therapies for Cystic Fibrosis Disease.

• Authors : Mancini G; Department of Medical Biotechnology and Translational Medicine, University of Milano, LITA, Via Fratelli Cervi 93, 20090 Segrate, Milano, Italy.; Loberto N

Subjects: Adjuvants, Immunologic/Adjuvants, Immunologic/Adjuvants, Immunologic/*pharmacology ; Aminophenols/Aminophenols/Aminophenols/*pharmacology ; Aminopyridines/Aminopyridines/Aminopyridines/*pharmacology

• Source: International journal of molecular sciences [Int J Mol Sci] 2020 Jun 24; Vol. 21 (12). Date of Electronic Publication: 2020 Jun 24.Publisher: MDPI Country of Publication: Switzerland NLM ID: 101092791 Publication Model: Electronic Cited Medium: Internet ISSN: 1422-0067

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Prolonged co-treatment with HGF sustains epithelial integrity and improves pharmacological rescue of Phe508del-CFTR.

• Authors : Matos AM; Department of Human Genetics, National Health Institute 'Dr. Ricardo Jorge', Av. Padre Cruz, 1649-016, Lisboa, Portugal.; University of Lisboa, Faculty of Sciences, BioISI - Biosystems & Integrative Sciences Institute, Campo Grande - C8, 1749-016, Lisboa, Portugal.

Subjects: Aminophenols/Aminophenols/Aminophenols/*administration & dosage ; Aminopyridines/Aminopyridines/Aminopyridines/*administration & dosage ; Benzodioxoles/Benzodioxoles/Benzodioxoles/*administration & dosage

• Source: Scientific reports [Sci Rep] 2018 Aug 29; Vol. 8 (1), pp. 13026. Date of Electronic Publication: 2018 Aug 29.Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN:

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Lumacaftor/ivacaftor combination therapy for cystic fibrosis: A nationwide survey among clinicians.

• Authors : Casciaro R; IRCCS G. Gaslini Institute, Via Gerolamo Gaslini, Genova, Italy.; Costa S

Subjects: Aminophenols/Aminophenols/Aminophenols/*therapeutic use ; Aminopyridines/Aminopyridines/Aminopyridines/*therapeutic use ; Benzodioxoles/Benzodioxoles/Benzodioxoles/*therapeutic use

• Source: The clinical respiratory journal [Clin Respir J] 2018 Apr; Vol. 12 (4), pp. 1767-1768. Date of Electronic Publication: 2017 Aug 04.Publisher: Blackwell Publishing Country of Publication: England NLM ID: 101315570 Publication Model: Print-Electronic Cited Medium: Internet ISSN:

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CFTR modulator therapy for cystic fibrosis caused by the rare c.3700A>G mutation

• Authors : Phuan, Puay-Wah; Haggie, Peter M; Tan, Joseph A

Subjects: Medical Physiology; Biomedical and Clinical Sciences; Cystic Fibrosis

• Source: Journal of Cystic Fibrosis. 20(3)

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The effectiveness and value of novel treatments for cystic fibrosis.

• Authors : Kuntz, Karen; Wherry, Kael; Seidner, Matt

Subjects: Adolescent; Aminophenols; Aminopyridines

• Source: Journal of Managed Care and Specialty Pharmacy. 27(2)

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Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled, phase 3 trial

• Authors : Ratjen, F; Hug, C; Marigowda, G

Subjects: Science & Technology; Life Sciences & Biomedicine; Critical Care Medicine

• Source: 567 ; 557

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Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene

• Authors : Mario Renda; Paolo Scudieri; Luis J. V. Galietta ...

Subjects: chloride channel; Pyrrolidines; QH301-705.5

• Source: International Journal of Molecular SciencesVolume 22Issue 21International Journal of Molecular Sciences, Vol 22, Iss 11972, p 11972 (2021)

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Kystisen fibroosin uudistuva lääkehoito

• Authors : Malmivaara, Kirsi; Elenius, Varpu;

Subjects: Cystic Fibrosis; +drug therapy; +genetics

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