Analysis of cystic fibrosis-associated P67L CFTR illustrates barriers to personalized therapeutics for orphan diseases
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Orkambi® and amplifier co-therapy improves function from a rare CFTR mutation in gene-edited cells and patient tissue.Recent progress in translational cystic fibrosis research using precision medicine strategies.Ion channels of the lung and their role in disease pathogenesis.Toward inclusive therapy with CFTR modulators: Progress and challenges.Personalized Medicine in CF: From Modulator Development to Therapy for Cystic Fibrosis Patients with Rare CFTR Mutations.Transformative therapies for rare CFTR missense alleles.
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Analysis of cystic fibrosis-associated P67L CFTR illustrates barriers to personalized therapeutics for orphan diseases
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article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on September 2016
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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Analysis of cystic fibrosis-as ...... erapeutics for orphan diseases
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Analysis of cystic fibrosis-as ...... rapeutics for orphan diseases.
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type
label
Analysis of cystic fibrosis-as ...... erapeutics for orphan diseases
@en
Analysis of cystic fibrosis-as ...... rapeutics for orphan diseases.
@nl
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Analysis of cystic fibrosis-as ...... erapeutics for orphan diseases
@en
Analysis of cystic fibrosis-as ...... rapeutics for orphan diseases.
@nl
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Analysis of cystic fibrosis-as ...... erapeutics for orphan diseases
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Carleen M Sabusap
Carmel M McNicholas
Eric J Sorscher
James F Collawn
Jeong S Hong
Kevin L Kirk
Marina Mazur
W Joon Chung
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P356
10.1172/JCI.INSIGHT.86581
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2016-09-01T00:00:00Z