Scientists create digital twins to advance Pompe disease treatment

Scientists at Sanofi created digital counterparts of people with Pompe disease to support drug development and advance Pompe disease treatment. In a simulated clinical trial of digital twins with infantile-onset Pompe disease (IOPD), Sanofi’s Nexviazyme (avalglucosidase alfa), an enzyme replacement therapy, outperformed its first-generation Lumizyme (alglucosidase alfa)…

New Institute Aims to Leave No Rare Disease Patient Behind

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

Significance of Clinical Trials for Pompe Disease

Clinical trials can help researchers develop new treatments for disorders such as Pompe disease, which currently has no cure and few options for treating symptoms. Through these trials, scientists gather evidence to support the potential regulatory approval of new and better therapies that may improve the quality of…