A study to identify individuals at risk to be affected by late-onset Pompe disease who had previously been given a non-specific or tentative diagnosis for their muscle weakness (Pompe PURSUE).
Orphanet J Rare Dis. 2025 Jan 14;20(1):23. doi: 10.1186/s13023-024-03425-1.
Orphanet J Rare Dis. 2025.
PMID: 39810165
Free PMC article.