Evidence map›Paper›PMID 41583306›Full record

ArticleCureus2025

A Rare Co-occurrence of Duchenne Muscular Dystrophy and Glycerol Kinase Deficiency Associated With Xp21 Contiguous Gene Deletion Syndrome: A Case Report.

Lúcia Marques, Patrícia Lipari Pinto, Joana Coelho, Cristina Florindo, Mariana Soeiro E Sá, Raquel Rodrigues, Ana Sousa, Ana Gaspar

Abstract readCase Reports
In one paragraph

Article in Cureus, 2025. 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.

Lúcia MarquesDepartment of Pediatrics, Hospital Beatriz Ângelo, Lisbon, PRT.
Patrícia Lipari PintoHereditary Metabolic Disease Reference Center, Metabolic Unit, Department of Pediatrics, Santa Maria's Hospital, Lisbon North University Hospital Center, Entidade Pública Empresarial (EPE) Faculty of Medicine, University of Lisbon, Lisbon, PRT.
Joana CoelhoNeuropediatric Unit, Department of Pediatrics, Santa Maria's Hospital, Lisbon North University Hospital Center, Entidade Pública Empresarial (EPE) Faculty of Medicine, University of Lisbon, Lisbon, PRT.
Cristina FlorindoLaboratory of Metabolism and Genetics, Department of Pharmaceutical Sciences and Medicines, Faculty of Pharmacy, University of Lisbon, Lisbon, PRT.
Mariana Soeiro E SáDepartment of Medical Genetics, Santa Maria's Hospital, Lisbon North University Hospital Center, Entidade Pública Empresarial (EPE) Faculty of Medicine, University of Lisbon, Lisbon, PRT.
Raquel RodriguesDepartment of Medical Genetics, Santa Maria's Hospital, Lisbon North University Hospital Center, Entidade Pública Empresarial (EPE) Faculty of Medicine, University of Lisbon, Lisbon, PRT.
Ana SousaDepartment of Medical Genetics, Santa Maria's Hospital, Lisbon North University Hospital Center, Entidade Pública Empresarial (EPE) Faculty of Medicine, University of Lisbon, Lisbon, PRT.
Ana GasparHereditary Metabolic Disease Reference Center, Metabolic Unit, Department of Pediatrics, Santa Maria's Hospital, Lisbon North University Hospital Center, Entidade Pública Empresarial (EPE) Faculty of Medicine, University of Lisbon, Lisbon, PRT.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Glycerol kinase deficiency is an X-linked disorder and can occur in isolation or combined as part of the Xp21 continuous gene deletion syndrome. We report the first offspring of a non-consanguineous couple with this contiguous gene deletion syndrome. There was a family history of short stature and learning difficulties, and a personal history of two hospitalizations due to prostration, hypoglycemia, and metabolic acidosis, the first of which occurred at six months of age. The patient was referred to a neurodevelopment consultation due to a global developmental delay detected at two years of age and a genetic consultation at four years old. The array comparative genomic hybridization study identified a maternally inherited hemizygous deletion of the Xp21 region of approximately 6.08 Mb that included both Duchenne muscular dystrophy and glycerol kinase genes, confirming the diagnosis. The patient was referred to metabolic and neurology consultations. Motor examination revealed a waddling gait when running, calf hypertrophy, and a positive Gower's sign. Laboratory evaluation was notable for elevated creatine kinase, hyperglyceroluria, pseudohypertriglyceridemia, and increased transaminases. The patient had a normal adrenocorticotropic hormone stimulation test and normal aldosterone and renin levels. Currently, he has a multidisciplinary team follow-up, including therapies. He maintains deflazacort therapy and follows a nutrition plan based on a fat-restricted diet and avoidance of prolonged fasting to prevent further metabolic crises. This case highlights the importance of identifying the exact genetic defects, in addition to a global picture of symptoms. In our case, it was possible to diagnose complex kinase deficiency along with Duchenne muscular dystrophy. Consequently, it was optimal for multi-profile medical care accompanied by an adequate nutritional plan.

Indexed as

array-cghcomplex glycerol kinase deficiencycontiguous gene deletion syndromeduchenne muscular dystrophyintellectual disability

Identifiers

PMID41583306
PMCPMC12831191

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.