Evidence map›Paper›PMID 41671094›Full record

SynthesisJournal of neuromuscular diseases2026

The natural history of Becker muscular dystrophy: A systematic literature review.

Alexis T Mickle, Karissa M Johnston, Kristen L Ricchetti-Masterson, Andrew R Kennedy, Sarah Gc Korpach, Katherine L Gooch

3 registry-linked trialsAbstract readSystematic ReviewReview
In one paragraph

Synthesis in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 3 registered trials, which are not on this map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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.

NCT01070511 phase4completednot on this map

Functional Muscle Ischemia and PDE5A Inhibition in Becker Muscular Dystrophy

TypeinterventionalSponsorCedars-Sinai Medical CenterRan2010 to 2012Enrolled48ConditionsBecker Muscular DystrophyArmsTadalafil, Placebo
NCT01350154 phase2completednot on this map

Does Modulation of the nNOS System in Patients With Muscular Dystrophy and Defect nNOS Signalling Affect Cardiac, Muscular or Cognitive Function?

TypeinterventionalSponsorRigshospitalet, DenmarkRan2011 to 2013Enrolled17ConditionsBecker Muscular DystrophyArmsSildenafil, Placebo
NCT02147639 phase2 / phase3completednot on this map

Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy

TypeinterventionalSponsorCedars-Sinai Medical CenterRan2013 to 2014Enrolled19ConditionsBecker Muscular DystrophyArmsSodium Nitrate, Sodium Nitrate - double dose, Placebo, Increased exercise intensity
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

6 authors.

Alexis T MickleBroadstreet HEOR, Vancouver, BC, Canada.ORCID 0009-0004-2884-1167
Karissa M JohnstonBroadstreet HEOR, Vancouver, BC, Canada.ORCID 0000-0003-4570-9972
Kristen L Ricchetti-MastersonSarepta Therapeutics Inc., Cambridge, MA, USA.ORCID 0000-0001-6970-7045
Andrew R KennedyBroadstreet HEOR, Vancouver, BC, Canada.ORCID 0009-0009-0386-510X
Sarah Gc KorpachBroadstreet HEOR, Vancouver, BC, Canada.ORCID 0009-0009-2129-1007
Katherine L GoochSarepta Therapeutics Inc., Cambridge, MA, USA.ORCID 0009-0000-6382-614X

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundBecker muscular dystrophy (BMD) is caused primarily by in-frame mutations in the

methodsA systematic literature review (SLR) was refreshed in 2022 using MEDLINE and EMBASE to identify articles describing the natural history of BMD. The proportion of patients experiencing clinical milestones was reported by 'life-stage' age groups (0-17; 18-40; 41+ years) using patient-level data from the general BMD population; age at each milestone's occurrence as mean (standard deviation [SD]).

resultsFrom 4948 abstracts screened, 121 publications were included. Among 36 general BMD population studies, by age 41+ years (lifetime-risk proxy), 93.6% experienced muscle weakness; 69.4% cardiac involvement; 55.6% scoliosis; 47.4% loss of ambulation; and 33.3% ventilation. Decreased cognitive function or cognitive dysfunction were reported in 41% across all ages. Among those experiencing milestones (79 studies), mean (SD) age at symptom onset was 12.5 (9.7); muscle weakness, 19.9 (11.7); scoliosis, 24.9 (3.1); cardiac involvement, 31.9 (13.4); loss of ambulation, 33.3 (13.5); ventilation, 35.7 (12.2); and death at 55.6 (19.4) years. Data availability ranged from three to 1079 patients/outcome.

conclusionsThis SLR highlights the variability in disease presentation in BMD. Stratifying BMD populations into phenotype groups based on the full spectrum of clinical manifestations may better capture disease progression and enhance comparability across studies.Included clinical trials:ClinicalTrials.gov NCT01070511 (https://clinicaltrials.gov/study/NCT01070511), ClinicalTrials.gov NCT02147639 (https://clinicaltrials.gov/study/NCT02147639?term=Becker%20Muscular%20Dystrophy&intr=Sodium%20Nitrate&rank=3), ClinicalTrials.gov NCT01350154 and EudraCT number: 2010-024659-10 (https://clinicaltrials.gov/study/NCT01350154?term=NCT01350154&rank=1; https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-024659-10/results).

Indexed as

Muscular Dystrophy, DuchenneAge of OnsetDisease ProgressionHumansMuscle WeaknessPhenotypeBecker muscular dystrophydystrophinmuscular dystrophynatural historyrare diseasesystematic literature review

Identifiers

PMID41671094
PMCPMC13438641

What Socratic holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

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.