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Record W2132360332 · doi:10.1097/mcp.0b013e3282efbc56

New pulmonary therapies for cystic fibrosis

2007· review· en· W2132360332 on OpenAlexaff
Félix Ratjen

Bibliographic record

VenueCurrent Opinion in Pulmonary Medicine · 2007
Typereview
Languageen
FieldMedicine
TopicCystic Fibrosis Research Advances
Canadian institutionsSickKids FoundationHospital for Sick Children
Fundersnot available
KeywordsCystic fibrosisMedicineCystic fibrosis transmembrane conductance regulatorRegulatorClinical trialPharmacotherapyGenetic enhancementIntensive care medicineChloride channelBioinformaticsInternal medicineGeneBiology

Abstract

fetched live from OpenAlex

PURPOSE OF REVIEW: Improved knowledge of the molecular mechanisms underlying cystic fibrosis has led to a wide spectrum of new treatment approaches. This review addresses recent therapeutic strategies that either target the underlying defect or early steps in cystic fibrosis pathophysiology. RECENT FINDINGS: While gene therapy does not appear to be a therapeutic option in the near future, cystic fibrosis transmembrane regulator pharmacotherapy is currently being developed as an alternative to reduce cystic fibrosis transmembrane regulator degradation or improve its function. Two drugs that increase chloride secretion via an alternative chloride channel, Moli1901 and denufosol, have been shown to be safe in clinical studies that also suggested clinical efficacy. Osmotic therapy may be an alternative approach to increase airway surface liquid and is being studied as an early intervention strategy. SUMMARY: The spectrum of treatments for cystic fibrosis lung disease is rapidly increasing. While clinical efficacy for most of the compounds still has to be proven in large clinical trials, there is considerable hope that cystic fibrosis therapy will move from addressing the downstream sequelae of the cystic fibrosis transmembrane regulator defect to a more causal approach in the near future.

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: Other design · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.872
Threshold uncertainty score0.999

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.001
Meta-epidemiology (narrow)0.0010.001
Meta-epidemiology (broad)0.0040.001
Bibliometrics0.0020.002
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0010.000
Research integrity0.0010.002
Insufficient payload (model declined to judge)0.0010.000

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 head0.160
GPT teacher head0.468
Teacher spread0.308 · 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 designOther design
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

Citations28
Published2007
Admission routes1
Has abstractyes

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