A moss-derived type of enzyme replacement therapy (ERT) for Pompe disease may be more effective than the currently approved ERT in targeting muscle cells, according to a small study. Since the study focused on muscle cells grown in the lab, further research in a whole organism, such as a…
Myozyme
People with late-onset Pompe disease (LOPD) show significant increases in the amount of muscle replaced by fat while on treatment, which is linked to reduced muscle strength and motor function, according to a new study. These findings support the use of muscle imaging to assess…
The Muscular Dystrophy Association is set to host the first Pompe disease Patient-Focused Drug Development (PFDD) meeting next year to inform therapy developers, regulators, and other stakeholders about the experiences of Pompe disease patients and caregivers, as well as their expectations for future treatments. Patients and family members interested…
The use of enzyme replacement therapy is safe and effective in Japanese people living with Pompe disease, a new study shows. Titled “Long-Term Observation of the Safety and Effectiveness of Enzyme Replacement Therapy in Japanese Patients with Pompe Disease: Results From the Post-marketing Surveillance,” the study…
Long-term interruption of enzyme replacement therapy (ERT) can lead to negative outcomes for late-onset Pompe disease patients, affecting their lung and exercise capacity as well as quality of life, a Swiss retrospective study shows. However, for most patients, resuming ERT can help restore the key clinical parameters that deteriorated during the pause…
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Immunomodulation Plus Myozyme May Help Infants with Pompe Disease, But Seen to Carry Risks in Study
Adding immunomodulatory medicines to enzyme replacement therapy (ERT) — Myozyme (alglucosidase alfa) — gives temporary but not long-term protection from antibodies that can block ERT’s effectiveness in patients with classic infantile Pompe disease. Better immunomodulatory regimens are needed to induce long-term…
Results of a Phase 4 clinical trial support the effectiveness of Lumizyme (4,000 liters recombinant human GAA) for 52 weeks in maintaining clinical stability in Pompe disease patients previously treated with the therapy’s 160-liter formulation, called Myozyme. The study, “Efficacy, safety profile, and immunogenicity of alglucosidase alfa…