Evidence map›Paper›PMID 35620971›Full record

ReviewThe Journal of physiology2022

Breathing in Duchenne muscular dystrophy: translation to therapy.

Doreen Z Mhandire, David P Burns, Angela L Roger, Ken D O'Halloran, Mai K ElMallah

Open access · greenAbstract readReview
In one paragraph

Review in The Journal of physiology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.

0numbers the graph read from it
0cells of the map it votes in
17citing papers in PubMed
2.7field-weighted citation impact, top 9% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

17 citing papers in PubMed, 33 citations in OpenAlex.

  1. AAVcc47-The Yale journal of biology and medicine · 2026
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 2 institutions in 2 countries.

Doreen Z MhandireDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center Box 2644, Durham, NC, USA.
David P BurnsDepartment of Physiology, School of Medicine, College of Medicine & Health, University College Cork, Cork, Ireland.ORCID 0000-0001-7221-3628
Angela L RogerDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center Box 2644, Durham, NC, USA.
Ken D O'HalloranDepartment of Physiology, School of Medicine, College of Medicine & Health, University College Cork, Cork, Ireland.
Mai K ElMallahDivision of Pulmonary and Sleep Medicine, Department of Pediatrics, Duke University Medical Center Box 2644, Durham, NC, USA.ORCID 0000-0002-1775-1156
Duke Medical Center · USUniversity College Cork · IE

Funding

Targeted Therapy for Pompe DiseaseR01HD099486 · NICHD · DUKE UNIVERSITY · PI ELMALLAH, MAI · 2019 to 2023
$1.7M
Gene Therapy for Upper Airway and Respiratory Insufficiency in an ALS Mouse ModelR21NS098131 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ELMALLAH, MAI · 2016 to 2017
$447k
NICHD NIH HHS R01 HD099486NINDS NIH HHS R21 NS098131
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular disease caused by a deficiency in dystrophin - a structural protein which stabilises muscle during contraction. Dystrophin deficiency adversely affects the respiratory system leading to sleep-disordered breathing, hypoventilation, and weakness of the expiratory and inspiratory musculature, which culminate in severe respiratory dysfunction. Muscle degeneration-associated respiratory impairment in neuromuscular disease is a result of disruptions at multiple sites of the respiratory control network, including sensory and motor pathways. As a result of this pathology, respiratory failure is a leading cause of premature death in DMD patients. Currently available treatments for DMD respiratory insufficiency attenuate respiratory symptoms without completely reversing the underlying pathophysiology. This underscores the need to develop curative therapies to improve quality of life and longevity of DMD patients. This review summarises research findings on the pathophysiology of respiratory insufficiencies in DMD disease in humans and animal models, the clinical interventions available to ameliorate symptoms, and gene-based therapeutic strategies uncovered by preclinical animal studies.

Indexed as

Muscular Dystrophy, DuchenneNeuromuscular DiseasesAnimalsDisease Models, AnimalDystrophinHumansQuality of LifeRespirationDystrophinAAV gene therapycontrol of breathingDuchenne muscular dystrophy (DMD)dystrophinrespiratory insufficiencyrespiratory muscle

Identifiers

PMID35620971
PMCPMC9357048
OpenAlexW4281620605

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.