Evidence mapPaperPMID 41748841Full record

ArticleNeurology and therapy2026

Management of Duchenne Muscular Dystrophy in Clinical Practice: A Survey-Based Study in Spain.

Carlos Ortez, María Branas-Pampillón, Joaquín Alejandro Fernandez, Elisa Guillen, Ángeles Terrancle, Jorge Maurino, Marisol Montolio, Ana Camacho

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Article in Neurology and therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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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

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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

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No citing paper in PubMed yet.

4 · The record

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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

8 authors.

Carlos OrtezNeuromuscular Unit, Department of Neurology, Hospital Sant Joan de Deu, Barcelona, Spain.
María Branas-PampillónMedical Department, Roche Farma, Ribera del Loira 50, 28042, Madrid, Spain. maria.branas_pampillon@roche.com.
Joaquín Alejandro FernandezPediatric Neurology Unit, Hospital Reina Sofia, Cordoba, Spain.
Elisa GuillenMedical Department, Roche Farma, Ribera del Loira 50, 28042, Madrid, Spain.
Ángeles TerrancleMedical Department, Roche Farma, Ribera del Loira 50, 28042, Madrid, Spain.
Jorge MaurinoMedical Department, Roche Farma, Ribera del Loira 50, 28042, Madrid, Spain.
Marisol MontolioDuchenne Parent Project España, Madrid, Spain.
Ana CamachoPediatric Neurology Unit, Hospital Universitario, 12 de Octubre, Madrid, Spain.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionDuchenne muscular dystrophy (DMD) management is guided by established clinical standards, yet real-world implementation data in Spain remain limited. The DMD-NEEDS study aimed to characterize the pediatric management landscape as reported by a select cohort of specialists within the national pediatric neurology community.

methodsThis non-interventional, cross-sectional, web-based survey involved 41 pediatric neurologists from the Spanish Society of Pediatric Neurology (SENEP) who actively manage patients with DMD. The study focuses specifically on the pediatric stage and the transition to adult care.

resultsClinicians reported that a slight majority of patients (53.7%) were diagnosed between 2 and 3 years of age, frequently within 6 months of symptom onset. Multiplex ligation-dependent probe amplification (MLPA) was the primary diagnostic tool for 92.7% of participating clinicians, frequently supplemented by gene sequencing (80.5%) or genetic panels (75.6%) to confirm single point mutations and duplications. Estimated loss of ambulation typically occurred between ages 11 and 13 years, with major clinical complications emerging between ages 12 and 17 years. Adherence to pharmacological standards was high: 95.1% of respondents prescribed corticosteroids as first-line therapy, and 89.7% specifically utilized a high-dose deflazacort regimen (0.9 mg/kg/day). Despite these trends, 31.7% of respondents lacked access to multidisciplinary care teams, and 58.5% reported that health-related quality-of-life questionnaires are not utilized in routine practice.

conclusionThese findings reveal a proactive clinical landscape characterized by early diagnosis and high adherence to international pharmacological standards. However, persistent gaps in multidisciplinary access and patient-centered monitoring highlight critical unmet needs. The implementation of a dedicated multidisciplinary framework and standardized management protocols is essential for optimizing the care of patients with DMD.

Indexed as

CorticosteroidsDuchenne muscular dystrophyEarly diagnosisMultidisciplinary carePediatric neurologyQuality of life

Identifiers

PMID41748841
PMCPMC13172134

What Socratic holds

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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.