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Pediatric Drugs

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01.12.2018 | Review Article

Potentiators and Correctors in Paediatric Cystic Fibrosis Patients: A Narrative Review

verfasst von: R. Dobra, C. Edmondson, D. Hughes, I. Martin, J. C. Davies

Erschienen in: Pediatric Drugs | Ausgabe 6/2018

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Abstract

Cystic fibrosis is the most common inherited condition in the Caucasian population and is associated with significantly reduced life expectancy. Recent advances in treatment have focussed on addressing the underlying cause of the condition, the defective production, expression and function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Several drugs with different modes of action have produced promising results in clinical trials, and some have been incorporated into routine clinical care for specific patients in many countries worldwide. Further trials continue to explore the safety and efficacy of these drugs in the youngest age groups and to search for more effective therapies to treat the most common disease-causing gene mutations in an ever-expanding drug pipeline. As evidence mounts for the early onset of disease in young patients, the prospect of introducing disease-modifying therapy in early life becomes more pertinent, although the cost implications of these expensive drugs are significant. In this review, we summarise these new therapy advances and review those currently being explored in clinical trials.

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Titel: Potentiators and Correctors in Paediatric Cystic Fibrosis Patients: A Narrative Review
verfasst von: R. Dobra
C. Edmondson
D. Hughes
I. Martin
J. C. Davies
Publikationsdatum: 01.12.2018
Verlag: Springer International Publishing
Erschienen in: Pediatric Drugs / Ausgabe 6/2018
Print ISSN: 1174-5878
Elektronische ISSN: 1179-2019
DOI: https://doi.org/10.1007/s40272-018-0315-z

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Zwar scheint es laut einer Studie aus den USA und Kanada bei der Reanimation von Kindern außerhalb einer Klinik keinen Unterschied für das Überleben zu machen, ob die Wiederbelebungsmaßnahmen während des Transports in die Klinik stattfinden oder vor Ort ausgeführt werden. Jedoch gibt es dabei einige Einschränkungen und eine wichtige Ausnahme.

Alter der Mutter beeinflusst Risiko für kongenitale Anomalie

28.05.2024 Kinder- und Jugendgynäkologie Nachrichten

Welchen Einfluss das Alter ihrer Mutter auf das Risiko hat, dass Kinder mit nicht chromosomal bedingter Malformation zur Welt kommen, hat eine ungarische Studie untersucht. Sie zeigt: Nicht nur fortgeschrittenes Alter ist riskant.

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Springer Medizin

Abstract

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An Update in Antimicrobial Therapies and Infection Prevention in Pediatric Lung Transplant Recipients

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Acknowledgement to Referees

Sexually Transmitted Infections in Pregnant Women: Integrating Screening and Treatment into Prenatal Care