Parents face significant anxiety and uncertainty after their newborn screens positive for a lysosomal storage disorder (LSD) like Pompe…
Articles by Steve Bryson, PhD
A new noninvasive imaging tool called quantitative muscle ultrasound, or QMUS, can detect progressive muscle disease in people with…
Switching to Nexviazyme (avalglucosidase alfa) was safe and effective in a 12-year-old boy with infantile-onset Pompe disease (IOPD)…
Switching from Lumizyme (alglucosidase alfa) to Nexviazyme (avalglucosidase alfa) stabilizes or improves lung function in people with…
Scientists at Sanofi have developed a new type of enzyme replacement therapy (ERT) that can clear excess glycogen…
Locations where antibodies bind to enzyme replacement therapy (ERT) and potentially reduce its effectiveness in treating Pompe disease can…
Orthodontic treatment successfully addressed speech and swallowing problems in a young girl with infantile-onset Pompe disease (IOPD), according to…
A 43-year-old woman who was experiencing breathing problems and muscle weakness was eventually diagnosed with late-onset Pompe disease (LOPD)…
Researchers defined a threshold for improvements in lung function that adults with late-onset Pompe disease (LOPD) perceive as clinically…
Switching from Lumizyme (alglucosidase alfa) to Pombiliti + Opfolda (cipaglucosidase alfa and miglustat) improved or stabilized motor function,…