Associated Papers
WES-based screening of 7,000 newborns: A pilot study in Russia.
Shubina J et al.
Hgg Advances, 2024
39033325 (PubMed )
Supplement: mmc1.pdf
Late-Onset Pompe Disease with Normal Creatine Kinase Levels: The Importance of Rheumatological Suspicion.
Marotto D et al.
International Journal of Molecular Sciences, 2023
Gene Therapy for Pompe Disease: The Time is now.
Colella P et al.
Human Gene Therapy, 2019
Progress and challenges of gene therapy for Pompe disease.
Ronzitti G et al.
Annals of Translational Medicine, 2019
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