“The Pompe disease community has long awaited the development of a treatment that overcomes the limitations of enzyme replacement therapy. We are eager to see how well muscle-targeted GYS1 ...

At the moment Pompe disease is treated using enzyme replacement therapy, which is delivered via chronic intravenous infusions every two weeks, but according to Astellas suffers from poor take-up ...

Amicus Therapeutics benefits from strong revenue and limited competition. Learn why FOLD stock is a BUY, with a favorable ...

Sanofi's Genzyme rare diseases division already sells another enzyme replacement therapy for Pompe disease based on the recombinant enzyme alglucosidase alfa – sold as Myozyme in Europe and ...

Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...

A key driver of market expansion is the increasing incidence of Pompe disease ... Nexviazyme, as an enzyme replacement therapy (ERT), plays a crucial role in reducing glycogen buildup, improving ...

Shionogi has concluded an exclusive global licence agreement for Maze Therapeutics’ MZE001, an oral glycogen synthase 1 (GYS1) inhibitor to treat Pompe disease. The agreement grants Shionogi the ...