In the first years of treatment, Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — can effectively increase…
Articles by Joana Carvalho, PhD
A chest MRI can be used to detect early signs of respiratory muscle weakness in people with Pompe disease,…
Deferoxamine, a medication normally used to remove excess iron and aluminum from the body, can counteract the effects of…
Sanofi Genzyme‘s application requesting the approval of avalglucosidase alfa, an investigational enzyme replacement therapy (ERT) for Pompe…
Scientists have created a new mouse model of Pompe disease that displays the severe respiratory impairments experienced by many…
Spark Therapeutics has resumed enrollment into two international clinical studies testing SPK-3006, the company’s investigational gene…
Children with classic infantile Pompe disease on higher and more frequent doses of Myozyme (alglucosidase alfa) live longer…
Avalglucosidase alfa, an investigational next-generation enzyme replacement therapy (ERT), leads to clinically meaningful improvements in respiratory muscle strength and…
When administered early and above its recommended dose, Myozyme (alglucosidase alfa, known as Lumizyme in the U.S.) may…
Increased awareness of late-onset Pompe disease (LOPD) is crucial to shorten the delay in diagnosis and start appropriate…