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Record W4410547805 · doi:10.1002/ppul.71113

Current Standards and Future Directions of Duchenne Muscular Dystrophy Respiratory Care: The PPMD Italy Meeting Report

2025· article· en· W4410547805 on OpenAlexaff
Fabrizio Racca, Jonathan D. Finder, Eugenio Mercuri, Andrea Vianello, Michelle Chatwin, Alessandro Amaddeo, Grazia Crescimanno, Federica Trucco, Daniel W. Sheehan, Luigi Barberis, Filippo Buccella, Jodi Wolff, Sherri L. Katz, David J. Birnkrant

Bibliographic record

VenuePediatric Pulmonology · 2025
Typearticle
Languageen
FieldMedicine
TopicRespiratory Support and Mechanisms
Canadian institutionsChildren's Hospital of Eastern Ontario
FundersParent Project Muscular Dystrophy
KeywordsMedicineDuchenne muscular dystrophyIntensive care medicineCardiorespiratory fitnessRespiratory careRespiratory failurePulmonary function testingRespiratory therapistMuscular dystrophyPhysical therapySurgeryInternal medicine

Abstract

fetched live from OpenAlex

OBJECTIVE: This report summarizes key discussions from the meeting "Current Standards and Future Directions of Respiratory Assessment and Management of Duchenne Muscular Dystrophy (DMD)," organized by Parent Project Muscular Dystrophy (PPMD) Italy and the United States to address current challenges and opportunities in DMD respiratory care. METHODS: The meeting brought together researchers, clinicians, and patient advocates who shared experiences, discussed advancements in DMD respiratory management, and identified areas of debate that require further research. RESULTS: The speakers emphasized routine assessment of pulmonary function and of breathing during sleep to achieve timely diagnosis of respiratory complications. Therapeutic discussions focused on airway clearance and assisted ventilation, highlighting noninvasive ventilation (NIV) as the preferred modality, even for advanced respiratory failure. The respiratory implications of new pharmacological therapies were discussed. The speakers endorsed the importance of cardiorespiratory outcomes in assessments of drug efficacy. To assess a drug's clinical impact and to define current respiratory phenotypes, the trajectory of the absolute value of forced vital capacity (FVC) was proposed as a potentially better parameter than FVC percent predicted, which is favored in current drug studies. In regard to management of acute respiratory failure and perioperative situations, standards of care and areas needing future research were identified. CONCLUSION: In this meeting, many points of consensus emerged, as well as areas requiring further research. The necessity to involve patients and their families in all aspects of respiratory care was emphasized, as well as the need for patient centered outcomes in medical decision making and research.

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)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.945
Threshold uncertainty score0.521

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.001
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
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 head0.008
GPT teacher head0.286
Teacher spread0.278 · 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.

The models applied no category: nothing in the taxonomy fit this work.
Study designNot applicable
Domainnot available
GenreEmpirical

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

Citations8
Published2025
Admission routes1
Has abstractyes

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