late-onset Pompe disease

Miglustat — a component of AT-GAA, an investigational therapy for late-onset Pompe disease (PD) — enhances the activity of the other component in AT-GAA, cipaglucosidase alfa, researchers report. These findings were presented at the 2021 MDA Virtual Clinical & Scientific Conference, in the poster “Enhancing Delivery…

If you have late-onset Pompe disease (LOPD) and are employed, or are considering employment, you’re likely going to need some workplace adjustments, particularly as the disorder progresses. Here’s some information about how Pompe disease might affect your work life. What is Pompe disease? Pompe disease is a debilitating…

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…

While the effects of late-onset Pompe disease on everyday life are far-reaching, there are steps you can take in your daily routine to help ease these difficulties and work around your limitations. It just takes some advance planning, a little bit of creativity, and perhaps some adaptive equipment.

If you have a chronic disease such as late-onset Pompe disease, a Japanese concept called shoshin may help you to perceive the world, and your disorder, in a different light. Based on Zen Buddhism, shoshin means “beginner’s mind.” What is Pompe disease? Pompe disease is a debilitating and…

Treatment with AT-GAA, an investigational therapy for late-onset Pompe disease, led to improvements in measures of physical and lung function in the Phase 3 clinical trial PROPEL, top-line data show. “Data from the PROPEL study demonstrate the potential to further improve motor and respiratory functions in patients with Pompe…

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…

Spark Therapeutics has dosed the first patient in the Phase 1/2 RESOLUTE clinical trial of SPK-3006, an experimental gene therapy for people with late-onset Pompe disease (LOPD). “Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and…

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…