Literature DB >> 31831338

Expanding access to CFTR modulators for rare mutations: The utility of n-of-1 trials.

Amalia S Magaret1, Nicole Mayer-Hamblett2, Donald VanDevanter3.   

Abstract

Entities:  

Year:  2019        PMID： 31831338     DOI： 10.1016/j.jcf.2019.11.011

Source DB:  PubMed          Journal:  J Cyst Fibros        ISSN： 1569-1993            Impact factor:   5.482


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  3 in total

Review 1.  Building global development strategies for cf therapeutics during a transitional cftr modulator era.

Authors:  N Mayer-Hamblett; S van Koningsbruggen-Rietschel; D P Nichols; D R VanDevanter; J C Davies; T Lee; A G Durmowicz; F Ratjen; M W Konstan; K Pearson; S C Bell; J P Clancy; J L Taylor-Cousar; K De Boeck; S H Donaldson; D G Downey; P A Flume; P Drevinek; C H Goss; I Fajac; A S Magaret; B S Quon; S M Singleton; J M VanDalfsen; G Z Retsch-Bogart
Journal:  J Cyst Fibros       Date:  2020-06-07       Impact factor: 5.482

2.  Exploring the Potential for Collaborative Use of an App-Based Platform for n-of-1 Trials Among Healthcare Professionals That Treat Patients With Insomnia.

Authors:  Jason R Bobe; Jessica K De Freitas; Benjamin S Glicksberg
Journal:  Front Psychiatry       Date:  2020-09-04       Impact factor: 4.157

3.  The impact of FDA and EMA regulatory decision-making process on the access to CFTR modulators for the treatment of cystic fibrosis.

Authors:  Enrico Costa; Silvia Girotti; Francesca Pauro; Hubert G M Leufkens; Marco Cipolli
Journal:  Orphanet J Rare Dis       Date:  2022-05-07       Impact factor: 4.303
  3 in total

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