treatment

In the first years of treatment, Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — can effectively increase physical endurance, and improve lung function and muscle strength in adults with late-onset Pompe disease (LOPD), a real-world study found. Yet, despite this treatment, patients’ clinical outcomes, particularly lung…

Early treatment with enzyme replacement therapy (ERT) and immune tolerance induction (ITI) greatly improved clinical outcomes for infants with the severe, frequently ERT-nonresponsive, CRIM-negative infantile Pompe disease (IPD), a study has found. “Our data suggest that the first few weeks after birth…

Patients with late-onset Pompe disease (LOPD) usually develop breathing problems as the disease progresses. If you have LOPD, you may eventually need ventilation to aid your breathing. Here is more information about how Pompe affects breathing and about ventilators that may help to ease the disease’s symptoms. How…

Lumizyme (alglucosidase alfa) is an enzyme replacement infusion therapy by Sanofi Genzyme for people with Pompe disease. In the European Union, it is available under the brand name Myozyme. Here are answers to some frequently asked questions about this treatment. How does Lumizyme work? Lumizyme works to replace…

Hearing loss is a very common for infants with Pompe disease. It may also affect some people who develop Pompe disease later in life. Here is information about how hearing works, how Pompe disease can cause problems with hearing, and how doctors diagnose and treat hearing loss in these patients.

Late-onset Pompe disease (LOPD) can affect a number of different organs and systems in the body. If you have the disorder, you will need to see a multidisciplinary treatment team to ensure you receive the best possible care. Read on for more information about some of the complications that…

Deferoxamine, a medication normally used to remove excess iron and aluminum from the body, can counteract the effects of a disease-causing genetic variant often found in people with late-onset Pompe disease (LOPD). These findings highlight the potential of drug repurposing for the development of new therapies for LOPD,…

If your child has been diagnosed with infantile-onset Pompe disease (IOPD), you may need a multidisciplinary team of specialists to ensure the best care possible. IOPD causes a buildup of glycogen (a large sugar molecule that the body uses to store energy), due to a lack or low levels…

If you have late-onset Pompe disease, you likely will need to see many specialists such as pulmonary physicians, cardiologists, and physiotherapists to help you manage the disorder’s different symptoms. To help with muscle weakness and other Pompe disease-related symptoms, it also may be beneficial to add a massage…

Amicus Therapeutics is seeking U.S. approval of AT-GAA, its investigational therapy for late-onset Pompe disease. The company has begun a rolling submission of a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA). This allows Amicus to submit individual sections of the application as soon they are…