![Astellas Gene Therapies on X: "#DYK Pompe disease is a severe, progressive, congenital neuromuscular disease caused by mutations in the acid alpha-glucosidase (GAA) gene? Join us this #InternationalPompeDay to help promote a Astellas Gene Therapies on X: "#DYK Pompe disease is a severe, progressive, congenital neuromuscular disease caused by mutations in the acid alpha-glucosidase (GAA) gene? Join us this #InternationalPompeDay to help promote a](https://pbs.twimg.com/ext_tw_video_thumb/1647592972874141697/pu/img/OmzPf-LZwcPxquNM.jpg:large)
Astellas Gene Therapies on X: "#DYK Pompe disease is a severe, progressive, congenital neuromuscular disease caused by mutations in the acid alpha-glucosidase (GAA) gene? Join us this #InternationalPompeDay to help promote a
![PPT - Pompe disease: a case study of the development of a successful treatment for a rare disease PowerPoint Presentation - ID:4054020 PPT - Pompe disease: a case study of the development of a successful treatment for a rare disease PowerPoint Presentation - ID:4054020](https://cdn2.slideserve.com/4054020/pompe-disease-a-case-study-of-the-development-of-a-successful-treatment-for-a-rare-disease-n.jpg)
PPT - Pompe disease: a case study of the development of a successful treatment for a rare disease PowerPoint Presentation - ID:4054020
![Pompe disease: a case study of the development of a successful treatment for a rare disease Kevin O'Donnell. - ppt download Pompe disease: a case study of the development of a successful treatment for a rare disease Kevin O'Donnell. - ppt download](https://images.slideplayer.com/24/7320302/slides/slide_2.jpg)