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Record W2092130790 · doi:10.1517/14728214.2010.517746

Emerging therapies for cystic fibrosis lung disease

2010· review· en· W2092130790 on OpenAlexaff
Hartmut Grasemann, Félix Ratjen

Bibliographic record

VenueExpert Opinion on Emerging Drugs · 2010
Typereview
Languageen
FieldMedicine
TopicCystic Fibrosis Research Advances
Canadian institutionsHospital for Sick ChildrenSickKids FoundationUniversity of Toronto
Fundersnot available
KeywordsCystic fibrosisMucociliary clearanceMedicineChloride channelAirwayLungCystic fibrosis transmembrane conductance regulatorFibrosisRespiratory systemLung diseaseIon channelPathologyInternal medicineReceptorCell biologyAnesthesiaBiology

Abstract

fetched live from OpenAlex

IMPORTANCE OF THE FIELD: Cystic fibrosis (CF), one of the major respiratory diseases, is caused by mutations in a gene encoding for a chloride channel. Abnormal transepithelial ion transport leads to a reduced volume of the airway surface liquid layer and reduced mucociliary clearance. AREAS COVERED IN THIS REVIEW: There is currently no cure for CF and CF lung disease remains a major contributor to morbidity and mortality. However, most current treatments for CF lung disease do not address the underlying pathology. We describe here new therapeutic developments aiming to identify or generate compounds that counteract the effects of cystic fibrosis transmembrane conductance regulator (CFTR) on the airway. WHAT THE READER WILL GAIN: This review summarizes the current state of new developments in the treatment of CF lung disease. These drugs include nebulized and inhaled osmotically active agents, but also modifiers of ion channels other than CFTR, such as activators of alternative chloride channels or inhibitors of sodium absorption, and compounds in development aim to correct or improve impaired CFTR function directly. First clinical trials with new drugs including ion channel modifiers and CFTR pharmacotherapeutics have revealed very promising results. TAKE HOME MESSAGE: CF drug therapy is moving rapidly from symptomatic therapy to treatment of the underlying pathophysiology.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.001
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.963
Threshold uncertainty score0.999

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.001
Meta-epidemiology (narrow)0.0010.001
Meta-epidemiology (broad)0.0020.001
Bibliometrics0.0010.001
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0010.000
Research integrity0.0000.001
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.035
GPT teacher head0.410
Teacher spread0.375 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

Study designNot applicable
Domainnot available
GenreReview

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations32
Published2010
Admission routes1
Has abstractyes

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