Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development Article Swipe
Thomas Desmet
,
Lauren Van Haesendonck
,
Séverine Vermeire
,
Eline van Overbeeke
,
Steven Simoens
,
Isabelle Huys
·
YOU?
·
· 2025
· Open Access
·
· DOI: https://doi.org/10.3389/fphar.2025.1662586
YOU?
·
· 2025
· Open Access
·
· DOI: https://doi.org/10.3389/fphar.2025.1662586
Background Duchenne muscular dystrophy (DMD) is an X-linked degenerative muscle disease with no curative treatment available to date. The current long-term use of corticosteroids is associated with severe adverse effects. With the progress of promising gene therapy for DMD, this research aims to identify the key characteristics that matter most to patients, develop attributes for a subsequent quantitative preference study, ultimately aimed to inform future market access and clinical decision-making on gene therapy. Methods A literature review was conducted, followed by semi-structured interviews with DMD patients and caregivers to explore their preferences regarding DMD treatment benefits and side effects and gene therapy as a promising treatment option. A ranking exercise helped reveal the most important treatment characteristics, forming the basis for the first step of a structured, four-step attribute and level development process: (1) attribute identification, (2) attribute selection, (3) attribute description, and (4) level development. The forthcoming six attributes and levels were determined by applying six inclusion and exclusion criteria aligned with PREFER guidelines and reaching consensus within an international multidisciplinary advisory board comprising patient representatives, clinicians, and preference method experts. Results A total of thirteen interviews were conducted with seven DMD patients and eleven caregivers. The literature review and interviews resulted in the identification of 48 unique disease and treatment characteristics. Furthermore, they revealed a high willingness of caregivers of especially younger children to consider gene therapy in a clinical trial setting, and that the primary treatment characteristics valued by patients and caregivers are related to muscle and heart function, and the impact on self-care activities, independence. The final attributes are patient-friendly, clinically relevant and meaningful to patients, with descriptions that are as brief as possible: the type of therapy, effect on life expectancy, risk of life-threatening side effects related to the therapy, years that ventilatory support can be postponed, number of years maintaining current physical functioning, and years and number of patients in which that therapy has been studied. Conclusion This study identified the treatment characteristics most important to DMD patients and their caregivers and translated them into six key attributes with corresponding levels. It underscores the practical value of qualitative research and patient engagement in ensuring that attributes and level development for future quantitative preference elicitation studies remain clinically relevant and aligned with patient priorities.
Related Topics
Concepts
No concepts available.
Metadata
- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.3389/fphar.2025.1662586
- https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1662586/pdf
- OA Status
- gold
- References
- 75
- OpenAlex ID
- https://openalex.org/W4414618427
All OpenAlex metadata
Raw OpenAlex JSON
- OpenAlex ID
-
https://openalex.org/W4414618427Canonical identifier for this work in OpenAlex
- DOI
-
https://doi.org/10.3389/fphar.2025.1662586Digital Object Identifier
- Title
-
Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute developmentWork title
- Type
-
articleOpenAlex work type
- Language
-
enPrimary language
- Publication year
-
2025Year of publication
- Publication date
-
2025-09-29Full publication date if available
- Authors
-
Thomas Desmet, Lauren Van Haesendonck, Séverine Vermeire, Eline van Overbeeke, Steven Simoens, Isabelle HuysList of authors in order
- Landing page
-
https://doi.org/10.3389/fphar.2025.1662586Publisher landing page
- PDF URL
-
https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1662586/pdfDirect link to full text PDF
- Open access
-
YesWhether a free full text is available
- OA status
-
goldOpen access status per OpenAlex
- OA URL
-
https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1662586/pdfDirect OA link when available
- Cited by
-
0Total citation count in OpenAlex
- References (count)
-
75Number of works referenced by this work
Full payload
| id | https://openalex.org/W4414618427 |
|---|---|
| doi | https://doi.org/10.3389/fphar.2025.1662586 |
| ids.doi | https://doi.org/10.3389/fphar.2025.1662586 |
| ids.pmid | https://pubmed.ncbi.nlm.nih.gov/41089854 |
| ids.openalex | https://openalex.org/W4414618427 |
| fwci | 0.0 |
| type | article |
| title | Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development |
| biblio.issue | |
| biblio.volume | 16 |
| biblio.last_page | 1662586 |
| biblio.first_page | 1662586 |
| topics[0].id | https://openalex.org/T10441 |
| topics[0].field.id | https://openalex.org/fields/13 |
| topics[0].field.display_name | Biochemistry, Genetics and Molecular Biology |
| topics[0].score | 0.9965999722480774 |
| topics[0].domain.id | https://openalex.org/domains/1 |
| topics[0].domain.display_name | Life Sciences |
| topics[0].subfield.id | https://openalex.org/subfields/1312 |
| topics[0].subfield.display_name | Molecular Biology |
| topics[0].display_name | Muscle Physiology and Disorders |
| topics[1].id | https://openalex.org/T10613 |
| topics[1].field.id | https://openalex.org/fields/13 |
| topics[1].field.display_name | Biochemistry, Genetics and Molecular Biology |
| topics[1].score | 0.9717000126838684 |
| topics[1].domain.id | https://openalex.org/domains/1 |
| topics[1].domain.display_name | Life Sciences |
| topics[1].subfield.id | https://openalex.org/subfields/1311 |
| topics[1].subfield.display_name | Genetics |
| topics[1].display_name | Virus-based gene therapy research |
| topics[2].id | https://openalex.org/T10106 |
| topics[2].field.id | https://openalex.org/fields/28 |
| topics[2].field.display_name | Neuroscience |
| topics[2].score | 0.9535999894142151 |
| topics[2].domain.id | https://openalex.org/domains/1 |
| topics[2].domain.display_name | Life Sciences |
| topics[2].subfield.id | https://openalex.org/subfields/2805 |
| topics[2].subfield.display_name | Cognitive Neuroscience |
| topics[2].display_name | Autism Spectrum Disorder Research |
| is_xpac | False |
| apc_list.value | 2950 |
| apc_list.currency | USD |
| apc_list.value_usd | 2950 |
| apc_paid.value | 2950 |
| apc_paid.currency | USD |
| apc_paid.value_usd | 2950 |
| language | en |
| locations[0].id | doi:10.3389/fphar.2025.1662586 |
| locations[0].is_oa | True |
| locations[0].source.id | https://openalex.org/S132108250 |
| locations[0].source.issn | 1663-9812 |
| locations[0].source.type | journal |
| locations[0].source.is_oa | True |
| locations[0].source.issn_l | 1663-9812 |
| locations[0].source.is_core | True |
| locations[0].source.is_in_doaj | True |
| locations[0].source.display_name | Frontiers in Pharmacology |
| locations[0].source.host_organization | https://openalex.org/P4310320527 |
| locations[0].source.host_organization_name | Frontiers Media |
| locations[0].source.host_organization_lineage | https://openalex.org/P4310320527 |
| locations[0].license | cc-by |
| locations[0].pdf_url | https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1662586/pdf |
| locations[0].version | publishedVersion |
| locations[0].raw_type | journal-article |
| locations[0].license_id | https://openalex.org/licenses/cc-by |
| locations[0].is_accepted | True |
| locations[0].is_published | True |
| locations[0].raw_source_name | Frontiers in Pharmacology |
| locations[0].landing_page_url | https://doi.org/10.3389/fphar.2025.1662586 |
| locations[1].id | pmid:41089854 |
| locations[1].is_oa | False |
| locations[1].source.id | https://openalex.org/S4306525036 |
| locations[1].source.issn | |
| locations[1].source.type | repository |
| locations[1].source.is_oa | False |
| locations[1].source.issn_l | |
| locations[1].source.is_core | False |
| locations[1].source.is_in_doaj | False |
| locations[1].source.display_name | PubMed |
| locations[1].source.host_organization | https://openalex.org/I1299303238 |
| locations[1].source.host_organization_name | National Institutes of Health |
| locations[1].source.host_organization_lineage | https://openalex.org/I1299303238 |
| locations[1].license | |
| locations[1].pdf_url | |
| locations[1].version | publishedVersion |
| locations[1].raw_type | |
| locations[1].license_id | |
| locations[1].is_accepted | True |
| locations[1].is_published | True |
| locations[1].raw_source_name | Frontiers in pharmacology |
| locations[1].landing_page_url | https://pubmed.ncbi.nlm.nih.gov/41089854 |
| locations[2].id | pmh:oai:doaj.org/article:d171a126e11e460587e2bdfd314571fa |
| locations[2].is_oa | False |
| locations[2].source.id | https://openalex.org/S4306401280 |
| locations[2].source.issn | |
| locations[2].source.type | repository |
| locations[2].source.is_oa | False |
| locations[2].source.issn_l | |
| locations[2].source.is_core | False |
| locations[2].source.is_in_doaj | False |
| locations[2].source.display_name | DOAJ (DOAJ: Directory of Open Access Journals) |
| locations[2].source.host_organization | |
| locations[2].source.host_organization_name | |
| locations[2].source.host_organization_lineage | |
| locations[2].license | |
| locations[2].pdf_url | |
| locations[2].version | submittedVersion |
| locations[2].raw_type | article |
| locations[2].license_id | |
| locations[2].is_accepted | False |
| locations[2].is_published | False |
| locations[2].raw_source_name | Frontiers in Pharmacology, Vol 16 (2025) |
| locations[2].landing_page_url | https://doaj.org/article/d171a126e11e460587e2bdfd314571fa |
| locations[3].id | pmh:oai:europepmc.org:11323416 |
| locations[3].is_oa | True |
| locations[3].source.id | https://openalex.org/S4306400806 |
| locations[3].source.issn | |
| locations[3].source.type | repository |
| locations[3].source.is_oa | False |
| locations[3].source.issn_l | |
| locations[3].source.is_core | False |
| locations[3].source.is_in_doaj | False |
| locations[3].source.display_name | Europe PMC (PubMed Central) |
| locations[3].source.host_organization | https://openalex.org/I1303153112 |
| locations[3].source.host_organization_name | European Bioinformatics Institute |
| locations[3].source.host_organization_lineage | https://openalex.org/I1303153112 |
| locations[3].license | other-oa |
| locations[3].pdf_url | |
| locations[3].version | submittedVersion |
| locations[3].raw_type | Text |
| locations[3].license_id | https://openalex.org/licenses/other-oa |
| locations[3].is_accepted | False |
| locations[3].is_published | False |
| locations[3].raw_source_name | |
| locations[3].landing_page_url | https://www.ncbi.nlm.nih.gov/pmc/articles/12515650 |
| indexed_in | crossref, doaj, pubmed |
| authorships[0].author.id | https://openalex.org/A5036910511 |
| authorships[0].author.orcid | https://orcid.org/0000-0001-7630-4850 |
| authorships[0].author.display_name | Thomas Desmet |
| authorships[0].countries | BE |
| authorships[0].affiliations[0].institution_ids | https://openalex.org/I99464096 |
| authorships[0].affiliations[0].raw_affiliation_string | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[0].affiliations[1].institution_ids | https://openalex.org/I4210160754 |
| authorships[0].affiliations[1].raw_affiliation_string | Healthcare Management Centre, Vlerick Business School, Ghent, Belgium |
| authorships[0].institutions[0].id | https://openalex.org/I99464096 |
| authorships[0].institutions[0].ror | https://ror.org/05f950310 |
| authorships[0].institutions[0].type | education |
| authorships[0].institutions[0].lineage | https://openalex.org/I99464096 |
| authorships[0].institutions[0].country_code | BE |
| authorships[0].institutions[0].display_name | KU Leuven |
| authorships[0].institutions[1].id | https://openalex.org/I4210160754 |
| authorships[0].institutions[1].ror | https://ror.org/056t38c37 |
| authorships[0].institutions[1].type | education |
| authorships[0].institutions[1].lineage | https://openalex.org/I4210160754 |
| authorships[0].institutions[1].country_code | BE |
| authorships[0].institutions[1].display_name | Vlerick Business School |
| authorships[0].author_position | first |
| authorships[0].raw_author_name | Thomas Desmet |
| authorships[0].is_corresponding | True |
| authorships[0].raw_affiliation_strings | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium, Healthcare Management Centre, Vlerick Business School, Ghent, Belgium |
| authorships[1].author.id | https://openalex.org/A5003443719 |
| authorships[1].author.orcid | |
| authorships[1].author.display_name | Lauren Van Haesendonck |
| authorships[1].countries | BE |
| authorships[1].affiliations[0].institution_ids | https://openalex.org/I99464096 |
| authorships[1].affiliations[0].raw_affiliation_string | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[1].institutions[0].id | https://openalex.org/I99464096 |
| authorships[1].institutions[0].ror | https://ror.org/05f950310 |
| authorships[1].institutions[0].type | education |
| authorships[1].institutions[0].lineage | https://openalex.org/I99464096 |
| authorships[1].institutions[0].country_code | BE |
| authorships[1].institutions[0].display_name | KU Leuven |
| authorships[1].author_position | middle |
| authorships[1].raw_author_name | Lauren Van Haesendonck |
| authorships[1].is_corresponding | False |
| authorships[1].raw_affiliation_strings | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[2].author.id | https://openalex.org/A5019474609 |
| authorships[2].author.orcid | https://orcid.org/0000-0001-9942-3019 |
| authorships[2].author.display_name | Séverine Vermeire |
| authorships[2].countries | BE |
| authorships[2].affiliations[0].institution_ids | https://openalex.org/I99464096 |
| authorships[2].affiliations[0].raw_affiliation_string | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[2].institutions[0].id | https://openalex.org/I99464096 |
| authorships[2].institutions[0].ror | https://ror.org/05f950310 |
| authorships[2].institutions[0].type | education |
| authorships[2].institutions[0].lineage | https://openalex.org/I99464096 |
| authorships[2].institutions[0].country_code | BE |
| authorships[2].institutions[0].display_name | KU Leuven |
| authorships[2].author_position | middle |
| authorships[2].raw_author_name | Sophie Vermeire |
| authorships[2].is_corresponding | False |
| authorships[2].raw_affiliation_strings | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[3].author.id | https://openalex.org/A5030479441 |
| authorships[3].author.orcid | https://orcid.org/0000-0003-0073-9350 |
| authorships[3].author.display_name | Eline van Overbeeke |
| authorships[3].countries | BE |
| authorships[3].affiliations[0].institution_ids | https://openalex.org/I99464096 |
| authorships[3].affiliations[0].raw_affiliation_string | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[3].institutions[0].id | https://openalex.org/I99464096 |
| authorships[3].institutions[0].ror | https://ror.org/05f950310 |
| authorships[3].institutions[0].type | education |
| authorships[3].institutions[0].lineage | https://openalex.org/I99464096 |
| authorships[3].institutions[0].country_code | BE |
| authorships[3].institutions[0].display_name | KU Leuven |
| authorships[3].author_position | middle |
| authorships[3].raw_author_name | Eline van Overbeeke |
| authorships[3].is_corresponding | False |
| authorships[3].raw_affiliation_strings | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[4].author.id | https://openalex.org/A5047342188 |
| authorships[4].author.orcid | https://orcid.org/0000-0002-9512-2005 |
| authorships[4].author.display_name | Steven Simoens |
| authorships[4].countries | BE |
| authorships[4].affiliations[0].institution_ids | https://openalex.org/I99464096 |
| authorships[4].affiliations[0].raw_affiliation_string | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[4].institutions[0].id | https://openalex.org/I99464096 |
| authorships[4].institutions[0].ror | https://ror.org/05f950310 |
| authorships[4].institutions[0].type | education |
| authorships[4].institutions[0].lineage | https://openalex.org/I99464096 |
| authorships[4].institutions[0].country_code | BE |
| authorships[4].institutions[0].display_name | KU Leuven |
| authorships[4].author_position | middle |
| authorships[4].raw_author_name | Steven Simoens |
| authorships[4].is_corresponding | False |
| authorships[4].raw_affiliation_strings | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[5].author.id | https://openalex.org/A5082978640 |
| authorships[5].author.orcid | https://orcid.org/0000-0002-4738-8298 |
| authorships[5].author.display_name | Isabelle Huys |
| authorships[5].countries | BE |
| authorships[5].affiliations[0].institution_ids | https://openalex.org/I99464096 |
| authorships[5].affiliations[0].raw_affiliation_string | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| authorships[5].institutions[0].id | https://openalex.org/I99464096 |
| authorships[5].institutions[0].ror | https://ror.org/05f950310 |
| authorships[5].institutions[0].type | education |
| authorships[5].institutions[0].lineage | https://openalex.org/I99464096 |
| authorships[5].institutions[0].country_code | BE |
| authorships[5].institutions[0].display_name | KU Leuven |
| authorships[5].author_position | last |
| authorships[5].raw_author_name | Isabelle Huys |
| authorships[5].is_corresponding | False |
| authorships[5].raw_affiliation_strings | Department of Pharmaceutical and Pharmacological Sciences, Clinical Pharmacology and Pharmacotherapy KU Leuven, Leuven, Belgium |
| has_content.pdf | True |
| has_content.grobid_xml | False |
| is_paratext | False |
| open_access.is_oa | True |
| open_access.oa_url | https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1662586/pdf |
| open_access.oa_status | gold |
| open_access.any_repository_has_fulltext | False |
| created_date | 2025-10-10T00:00:00 |
| display_name | Preferences for gene therapy in Duchenne muscular dystrophy: insights from patient and caregiver interviews and attribute development |
| has_fulltext | False |
| is_retracted | False |
| updated_date | 2025-11-06T03:46:38.306776 |
| primary_topic.id | https://openalex.org/T10441 |
| primary_topic.field.id | https://openalex.org/fields/13 |
| primary_topic.field.display_name | Biochemistry, Genetics and Molecular Biology |
| primary_topic.score | 0.9965999722480774 |
| primary_topic.domain.id | https://openalex.org/domains/1 |
| primary_topic.domain.display_name | Life Sciences |
| primary_topic.subfield.id | https://openalex.org/subfields/1312 |
| primary_topic.subfield.display_name | Molecular Biology |
| primary_topic.display_name | Muscle Physiology and Disorders |
| cited_by_count | 0 |
| locations_count | 4 |
| best_oa_location.id | doi:10.3389/fphar.2025.1662586 |
| best_oa_location.is_oa | True |
| best_oa_location.source.id | https://openalex.org/S132108250 |
| best_oa_location.source.issn | 1663-9812 |
| best_oa_location.source.type | journal |
| best_oa_location.source.is_oa | True |
| best_oa_location.source.issn_l | 1663-9812 |
| best_oa_location.source.is_core | True |
| best_oa_location.source.is_in_doaj | True |
| best_oa_location.source.display_name | Frontiers in Pharmacology |
| best_oa_location.source.host_organization | https://openalex.org/P4310320527 |
| best_oa_location.source.host_organization_name | Frontiers Media |
| best_oa_location.source.host_organization_lineage | https://openalex.org/P4310320527 |
| best_oa_location.license | cc-by |
| best_oa_location.pdf_url | https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1662586/pdf |
| best_oa_location.version | publishedVersion |
| best_oa_location.raw_type | journal-article |
| best_oa_location.license_id | https://openalex.org/licenses/cc-by |
| best_oa_location.is_accepted | True |
| best_oa_location.is_published | True |
| best_oa_location.raw_source_name | Frontiers in Pharmacology |
| best_oa_location.landing_page_url | https://doi.org/10.3389/fphar.2025.1662586 |
| primary_location.id | doi:10.3389/fphar.2025.1662586 |
| primary_location.is_oa | True |
| primary_location.source.id | https://openalex.org/S132108250 |
| primary_location.source.issn | 1663-9812 |
| primary_location.source.type | journal |
| primary_location.source.is_oa | True |
| primary_location.source.issn_l | 1663-9812 |
| primary_location.source.is_core | True |
| primary_location.source.is_in_doaj | True |
| primary_location.source.display_name | Frontiers in Pharmacology |
| primary_location.source.host_organization | https://openalex.org/P4310320527 |
| primary_location.source.host_organization_name | Frontiers Media |
| primary_location.source.host_organization_lineage | https://openalex.org/P4310320527 |
| primary_location.license | cc-by |
| primary_location.pdf_url | https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2025.1662586/pdf |
| primary_location.version | publishedVersion |
| primary_location.raw_type | journal-article |
| primary_location.license_id | https://openalex.org/licenses/cc-by |
| primary_location.is_accepted | True |
| primary_location.is_published | True |
| primary_location.raw_source_name | Frontiers in Pharmacology |
| primary_location.landing_page_url | https://doi.org/10.3389/fphar.2025.1662586 |
| publication_date | 2025-09-29 |
| publication_year | 2025 |
| referenced_works | https://openalex.org/W2079533829, https://openalex.org/W2803996799, https://openalex.org/W3111106831, https://openalex.org/W1489295247, https://openalex.org/W2494748207, https://openalex.org/W2969189244, https://openalex.org/W2883696211, https://openalex.org/W63642947, https://openalex.org/W2784812244, https://openalex.org/W2784992484, https://openalex.org/W2892657381, https://openalex.org/W2606840399, https://openalex.org/W2346705222, https://openalex.org/W1562592376, https://openalex.org/W3033757480, https://openalex.org/W2810938784, https://openalex.org/W4214892886, https://openalex.org/W2145106175, https://openalex.org/W2171854062, https://openalex.org/W2152842810, https://openalex.org/W1976770755, https://openalex.org/W2991696549, https://openalex.org/W1997745844, https://openalex.org/W2609113197, https://openalex.org/W2974120070, https://openalex.org/W3095800367, https://openalex.org/W2966204020, https://openalex.org/W2529732248, https://openalex.org/W2521883047, https://openalex.org/W2126800506, https://openalex.org/W2995066679, https://openalex.org/W1993475641, https://openalex.org/W2154417169, https://openalex.org/W2978655460, https://openalex.org/W3093682693, https://openalex.org/W2940758803, https://openalex.org/W2149395822, https://openalex.org/W3098825483, https://openalex.org/W2751682413, https://openalex.org/W2943189056, https://openalex.org/W2605991219, https://openalex.org/W2784803563, https://openalex.org/W2484125054, https://openalex.org/W1888941810, https://openalex.org/W2431474207, https://openalex.org/W2041857292, https://openalex.org/W2891193171, https://openalex.org/W4406615368, https://openalex.org/W2030785841, https://openalex.org/W2947503350, https://openalex.org/W2031257767, https://openalex.org/W2160653847, https://openalex.org/W2584905746, https://openalex.org/W2596498480, https://openalex.org/W2948946881, https://openalex.org/W2900078746, https://openalex.org/W2040380105, https://openalex.org/W2944141319, https://openalex.org/W2980523496, https://openalex.org/W2648365531, https://openalex.org/W4211211101, https://openalex.org/W2138664283, https://openalex.org/W3135253175, https://openalex.org/W2892818464, https://openalex.org/W3081946736, https://openalex.org/W3100971054, https://openalex.org/W1978943632, https://openalex.org/W2948019101, https://openalex.org/W2765526086, https://openalex.org/W1926939363, https://openalex.org/W4386995574, https://openalex.org/W2806907889, https://openalex.org/W3138386431, https://openalex.org/W1776608400, https://openalex.org/W2414531575 |
| referenced_works_count | 75 |
| abstract_inverted_index.A | 74, 107, 183 |
| abstract_inverted_index.a | 55, 103, 125, 216, 230 |
| abstract_inverted_index.48 | 207 |
| abstract_inverted_index.It | 347 |
| abstract_inverted_index.an | 6, 169 |
| abstract_inverted_index.as | 102, 274, 276 |
| abstract_inverted_index.be | 300 |
| abstract_inverted_index.by | 80, 154, 241 |
| abstract_inverted_index.in | 203, 229, 315, 358 |
| abstract_inverted_index.is | 5, 24 |
| abstract_inverted_index.no | 12 |
| abstract_inverted_index.of | 22, 33, 124, 185, 206, 219, 221, 280, 287, 303, 313, 352 |
| abstract_inverted_index.on | 70, 255, 283 |
| abstract_inverted_index.to | 16, 42, 50, 62, 88, 225, 247, 268, 292, 331 |
| abstract_inverted_index.(1) | 133 |
| abstract_inverted_index.(2) | 136 |
| abstract_inverted_index.(3) | 139 |
| abstract_inverted_index.(4) | 143 |
| abstract_inverted_index.DMD | 84, 93, 192, 332 |
| abstract_inverted_index.The | 18, 146, 197, 259 |
| abstract_inverted_index.and | 67, 86, 96, 99, 129, 142, 150, 158, 165, 178, 194, 200, 210, 234, 243, 249, 252, 266, 309, 311, 334, 337, 355, 362, 374 |
| abstract_inverted_index.are | 245, 262, 273 |
| abstract_inverted_index.can | 299 |
| abstract_inverted_index.for | 37, 54, 120, 365 |
| abstract_inverted_index.has | 319 |
| abstract_inverted_index.key | 45, 342 |
| abstract_inverted_index.six | 148, 156, 341 |
| abstract_inverted_index.the | 31, 44, 112, 118, 121, 204, 236, 253, 278, 293, 326, 349 |
| abstract_inverted_index.use | 21 |
| abstract_inverted_index.was | 77 |
| abstract_inverted_index.DMD, | 38 |
| abstract_inverted_index.This | 323 |
| abstract_inverted_index.With | 30 |
| abstract_inverted_index.aims | 41 |
| abstract_inverted_index.been | 320 |
| abstract_inverted_index.gene | 35, 71, 100, 227 |
| abstract_inverted_index.high | 217 |
| abstract_inverted_index.into | 340 |
| abstract_inverted_index.life | 284 |
| abstract_inverted_index.most | 49, 113, 329 |
| abstract_inverted_index.risk | 286 |
| abstract_inverted_index.side | 97, 289 |
| abstract_inverted_index.step | 123 |
| abstract_inverted_index.that | 47, 235, 272, 296, 317, 360 |
| abstract_inverted_index.them | 339 |
| abstract_inverted_index.they | 214 |
| abstract_inverted_index.this | 39 |
| abstract_inverted_index.type | 279 |
| abstract_inverted_index.were | 152, 188 |
| abstract_inverted_index.with | 11, 26, 83, 162, 190, 270, 344, 376 |
| abstract_inverted_index.(DMD) | 4 |
| abstract_inverted_index.aimed | 61 |
| abstract_inverted_index.basis | 119 |
| abstract_inverted_index.board | 173 |
| abstract_inverted_index.brief | 275 |
| abstract_inverted_index.date. | 17 |
| abstract_inverted_index.final | 260 |
| abstract_inverted_index.first | 122 |
| abstract_inverted_index.heart | 250 |
| abstract_inverted_index.level | 130, 144, 363 |
| abstract_inverted_index.seven | 191 |
| abstract_inverted_index.study | 324 |
| abstract_inverted_index.their | 90, 335 |
| abstract_inverted_index.total | 184 |
| abstract_inverted_index.trial | 232 |
| abstract_inverted_index.value | 351 |
| abstract_inverted_index.which | 316 |
| abstract_inverted_index.years | 295, 304, 310 |
| abstract_inverted_index.PREFER | 163 |
| abstract_inverted_index.access | 66 |
| abstract_inverted_index.effect | 282 |
| abstract_inverted_index.eleven | 195 |
| abstract_inverted_index.future | 64, 366 |
| abstract_inverted_index.helped | 110 |
| abstract_inverted_index.impact | 254 |
| abstract_inverted_index.inform | 63 |
| abstract_inverted_index.levels | 151 |
| abstract_inverted_index.market | 65 |
| abstract_inverted_index.matter | 48 |
| abstract_inverted_index.method | 180 |
| abstract_inverted_index.muscle | 9, 248 |
| abstract_inverted_index.number | 302, 312 |
| abstract_inverted_index.remain | 371 |
| abstract_inverted_index.reveal | 111 |
| abstract_inverted_index.review | 76, 199 |
| abstract_inverted_index.severe | 27 |
| abstract_inverted_index.study, | 59 |
| abstract_inverted_index.unique | 208 |
| abstract_inverted_index.valued | 240 |
| abstract_inverted_index.within | 168 |
| abstract_inverted_index.Methods | 73 |
| abstract_inverted_index.Results | 182 |
| abstract_inverted_index.adverse | 28 |
| abstract_inverted_index.aligned | 161, 375 |
| abstract_inverted_index.current | 19, 306 |
| abstract_inverted_index.develop | 52 |
| abstract_inverted_index.disease | 10, 209 |
| abstract_inverted_index.effects | 98, 290 |
| abstract_inverted_index.explore | 89 |
| abstract_inverted_index.forming | 117 |
| abstract_inverted_index.levels. | 346 |
| abstract_inverted_index.option. | 106 |
| abstract_inverted_index.patient | 175, 356, 377 |
| abstract_inverted_index.primary | 237 |
| abstract_inverted_index.ranking | 108 |
| abstract_inverted_index.related | 246, 291 |
| abstract_inverted_index.studies | 370 |
| abstract_inverted_index.support | 298 |
| abstract_inverted_index.therapy | 36, 101, 228, 318 |
| abstract_inverted_index.younger | 223 |
| abstract_inverted_index.Duchenne | 1 |
| abstract_inverted_index.X-linked | 7 |
| abstract_inverted_index.advisory | 172 |
| abstract_inverted_index.applying | 155 |
| abstract_inverted_index.benefits | 95 |
| abstract_inverted_index.children | 224 |
| abstract_inverted_index.clinical | 68, 231 |
| abstract_inverted_index.consider | 226 |
| abstract_inverted_index.criteria | 160 |
| abstract_inverted_index.curative | 13 |
| abstract_inverted_index.effects. | 29 |
| abstract_inverted_index.ensuring | 359 |
| abstract_inverted_index.exercise | 109 |
| abstract_inverted_index.experts. | 181 |
| abstract_inverted_index.followed | 79 |
| abstract_inverted_index.identify | 43 |
| abstract_inverted_index.muscular | 2 |
| abstract_inverted_index.patients | 85, 193, 242, 314, 333 |
| abstract_inverted_index.physical | 307 |
| abstract_inverted_index.process: | 132 |
| abstract_inverted_index.progress | 32 |
| abstract_inverted_index.reaching | 166 |
| abstract_inverted_index.relevant | 265, 373 |
| abstract_inverted_index.research | 40, 354 |
| abstract_inverted_index.resulted | 202 |
| abstract_inverted_index.revealed | 215 |
| abstract_inverted_index.setting, | 233 |
| abstract_inverted_index.studied. | 321 |
| abstract_inverted_index.therapy, | 281, 294 |
| abstract_inverted_index.therapy. | 72 |
| abstract_inverted_index.thirteen | 186 |
| abstract_inverted_index.attribute | 128, 134, 137, 140 |
| abstract_inverted_index.available | 15 |
| abstract_inverted_index.conducted | 189 |
| abstract_inverted_index.consensus | 167 |
| abstract_inverted_index.dystrophy | 3 |
| abstract_inverted_index.exclusion | 159 |
| abstract_inverted_index.four-step | 127 |
| abstract_inverted_index.function, | 251 |
| abstract_inverted_index.important | 114, 330 |
| abstract_inverted_index.inclusion | 157 |
| abstract_inverted_index.long-term | 20 |
| abstract_inverted_index.patients, | 51, 269 |
| abstract_inverted_index.possible: | 277 |
| abstract_inverted_index.practical | 350 |
| abstract_inverted_index.promising | 34, 104 |
| abstract_inverted_index.regarding | 92 |
| abstract_inverted_index.self-care | 256 |
| abstract_inverted_index.treatment | 14, 94, 105, 115, 211, 238, 327 |
| abstract_inverted_index.Background | 0 |
| abstract_inverted_index.Conclusion | 322 |
| abstract_inverted_index.associated | 25 |
| abstract_inverted_index.attributes | 53, 149, 261, 343, 361 |
| abstract_inverted_index.caregivers | 87, 220, 244, 336 |
| abstract_inverted_index.clinically | 264, 372 |
| abstract_inverted_index.comprising | 174 |
| abstract_inverted_index.conducted, | 78 |
| abstract_inverted_index.determined | 153 |
| abstract_inverted_index.engagement | 357 |
| abstract_inverted_index.especially | 222 |
| abstract_inverted_index.guidelines | 164 |
| abstract_inverted_index.identified | 325 |
| abstract_inverted_index.interviews | 82, 187, 201 |
| abstract_inverted_index.literature | 75, 198 |
| abstract_inverted_index.meaningful | 267 |
| abstract_inverted_index.postponed, | 301 |
| abstract_inverted_index.preference | 58, 179, 368 |
| abstract_inverted_index.selection, | 138 |
| abstract_inverted_index.subsequent | 56 |
| abstract_inverted_index.translated | 338 |
| abstract_inverted_index.ultimately | 60 |
| abstract_inverted_index.activities, | 257 |
| abstract_inverted_index.caregivers. | 196 |
| abstract_inverted_index.clinicians, | 177 |
| abstract_inverted_index.development | 131, 364 |
| abstract_inverted_index.elicitation | 369 |
| abstract_inverted_index.expectancy, | 285 |
| abstract_inverted_index.forthcoming | 147 |
| abstract_inverted_index.maintaining | 305 |
| abstract_inverted_index.preferences | 91 |
| abstract_inverted_index.priorities. | 378 |
| abstract_inverted_index.qualitative | 353 |
| abstract_inverted_index.structured, | 126 |
| abstract_inverted_index.underscores | 348 |
| abstract_inverted_index.ventilatory | 297 |
| abstract_inverted_index.willingness | 218 |
| abstract_inverted_index.Furthermore, | 213 |
| abstract_inverted_index.degenerative | 8 |
| abstract_inverted_index.description, | 141 |
| abstract_inverted_index.descriptions | 271 |
| abstract_inverted_index.development. | 145 |
| abstract_inverted_index.functioning, | 308 |
| abstract_inverted_index.quantitative | 57, 367 |
| abstract_inverted_index.corresponding | 345 |
| abstract_inverted_index.independence. | 258 |
| abstract_inverted_index.international | 170 |
| abstract_inverted_index.identification | 205 |
| abstract_inverted_index.characteristics | 46, 239, 328 |
| abstract_inverted_index.corticosteroids | 23 |
| abstract_inverted_index.decision-making | 69 |
| abstract_inverted_index.identification, | 135 |
| abstract_inverted_index.semi-structured | 81 |
| abstract_inverted_index.characteristics, | 116 |
| abstract_inverted_index.characteristics. | 212 |
| abstract_inverted_index.life-threatening | 288 |
| abstract_inverted_index.representatives, | 176 |
| abstract_inverted_index.multidisciplinary | 171 |
| abstract_inverted_index.patient-friendly, | 263 |
| cited_by_percentile_year | |
| corresponding_author_ids | https://openalex.org/A5036910511 |
| countries_distinct_count | 1 |
| institutions_distinct_count | 6 |
| corresponding_institution_ids | https://openalex.org/I4210160754, https://openalex.org/I99464096 |
| citation_normalized_percentile.value | 0.43792665 |
| citation_normalized_percentile.is_in_top_1_percent | False |
| citation_normalized_percentile.is_in_top_10_percent | False |