Case Report: Incidental late-onset Pompe disease diagnosis in a man with no clinical and instrumental evidence of neuromuscular dysfunction
Glycogen storage disease II or Pompe disease (PD), is a rare autosomal recessive disorder due to biallelic pathogenic variants in GAA, resulting in the enzymatic deficiency of alpha-1,4-glucosidase. Two clinical forms are recognized, namely, early onset (EOPD) and late-onset (LOPD). We present the c...
Saved in:
| Main Authors: | Monica Sciacco, Sabrina Lucchiari, Letizia Bertolasi, Giacomo Pietro Comi, Stefania Corti, Dario Ronchi |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2025-06-01
|
| Series: | Frontiers in Genetics |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fgene.2025.1574381/full |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
Late-onset Pompe disease: preliminary results of enzyme replacement therapy
by: L. P. Smertina, et al.
Published: (2019-07-01) -
Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA
by: Paolo Peruzzo, et al.
Published: (2025-02-01) -
Late-onset Pompe disease with phenotype of the limb-girdle muscular dystrophy
by: S. A. Kurbatov, et al.
Published: (2015-09-01) -
Global variations in diagnostic methods and epidemiological estimates in Pompe disease: findings from a scoping review
by: Roberto Giugliani, et al.
Published: (2025-05-01) -
Late-onset Pompe disease with complicated intracranial aneurysm: a Chinese case report
by: Zhang B, et al.
Published: (2016-03-01)