Evidence map›Paper›PMID 40853195›Full record

ReviewJournal of neuromuscular diseases2026

Rehabilitation research in spinal muscular atrophy: a call to action.

Charlotte Lilien, Leslie Nelson, Lisa Edel, Danielle Forrest, Timothy Estilow, Katlyn E McGrattan, Tina Duong, Giorgia Coratti

Abstract readReview
In one paragraph

Review in Journal of neuromuscular diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the 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.

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

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

8 authors.

Charlotte LilienSTRONG MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.ORCID 0009-0001-8219-6996
Leslie NelsonDepartment of Physical Therapy, University of Texas Southwestern Medical Center, Dallas, TX, USA.ORCID 0000-0003-3606-5757
Lisa EdelGreat Ormond Street Hospital, London, UK.ORCID 0000-0002-9352-5388
Danielle ForrestDepartment of Physical Therapy, University of Texas Southwestern Medical Center, Dallas, TX, USA.ORCID 0009-0009-4622-1232
Timothy EstilowDepartment of Occupational Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Katlyn E McGrattanDepartment of Speech Language and Hearing Science, University of Minnesota, Minneapolis, MN, USA.ORCID 0000-0002-7853-1624
Tina DuongJohn W. Day Lab, Department of Neurology and Neurological Sciences, University of Stanford, Stanford, USA.
Giorgia CorattiPediatric Neurology, Catholic University of Sacred Heart, Rome, Italy.ORCID 0000-0001-6666-5628

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

DesignThe landscape of spinal muscular atrophy drastically changed following the introduction of disease-modifying therapies, emphasizing the need for comprehensive rehabilitation strategies to maximize functional outcomes. Our aim is to identify the barriers faced by healthcare professionals in conducting rehabilitation research for spinal muscular atrophy and suggest potential solutions to increase evidence-based care.MethodsWe performed a narrative review of rehabilitation intervention studies with a quality assessment focusing on the research characteristics of each study and an international survey questioning healthcare professionals on their major areas of difficulty in conducting interventional studies.ResultsThe review highlighted a predominantly low to unacceptable quality of the 36 retained studies across four healthcare professional domains responsible for rehabilitation: physiotherapy/physical therapy (18), occupational therapy (4), respiratory therapy (10), and speech and language therapy/speech language pathology (4).The survey was completed by 204 health care providers from 35 countries. Funding was found to be the most significant barrier to rehabilitation research, followed by study design and recruitment challenges.ConclusionOur findings highlight the urgent need for randomized controlled trials and standardized methodologies to develop robust, evidence-based multi-disciplinary rehabilitation strategies that better support individuals with spinal muscular atrophy in achieving optimal functional outcomes in the era of disease-modifying treatments.

Indexed as

Muscular Atrophy, SpinalRehabilitation ResearchHumansPhysical Therapy ModalitiesbarrierschallengesrehabilitationresearchSMA

Identifiers

PMID40853195
PMCPMC13438077

What OpenQuestion holds

Textmetadata
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 OpenQuestion graph.