![Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium - The Lancet Child & Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium - The Lancet Child &](https://els-jbs-prod-cdn.jbs.elsevierhealth.com/cms/attachment/4eccf0b5-b460-4566-946d-79c3235032a0/gr1_lrg.jpg)
Effect of alglucosidase alfa dosage on survival and walking ability in patients with classic infantile Pompe disease: a multicentre observational cohort study from the European Pompe Consortium - The Lancet Child &
![Newborn screening for Pompe disease in Japan: report and literature review of mutations in the GAA gene in Japanese and Asian patients | Journal of Human Genetics Newborn screening for Pompe disease in Japan: report and literature review of mutations in the GAA gene in Japanese and Asian patients | Journal of Human Genetics](https://media.springernature.com/m685/springer-static/image/art%3A10.1038%2Fs10038-019-0603-7/MediaObjects/10038_2019_603_Fig1_HTML.png)
Newborn screening for Pompe disease in Japan: report and literature review of mutations in the GAA gene in Japanese and Asian patients | Journal of Human Genetics
![Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity | EMBO Molecular Medicine Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity | EMBO Molecular Medicine](https://www.embopress.org/cms/asset/21ed3ce1-e78a-40f7-92a4-e2374c12e956/emmm202113968-abs-0001-m.jpg)
Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity | EMBO Molecular Medicine
![Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease: Molecular Therapy - Methods & Clinical Development Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease: Molecular Therapy - Methods & Clinical Development](https://els-jbs-prod-cdn.jbs.elsevierhealth.com/cms/attachment/d53fd34d-530e-4e65-8202-1e841683219c/fx1_lrg.jpg)
Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease: Molecular Therapy - Methods & Clinical Development
![Gene Therapy for the Treatment of Pompe Disease | National Center for Advancing Translational Sciences Gene Therapy for the Treatment of Pompe Disease | National Center for Advancing Translational Sciences](https://ncats.nih.gov/files/trnd-pompe_900x600.jpg)