late-onset Pompe disease

The European Medicines Agency agreed to begin reviewing Sanofi Genzyme’s application requesting the approval of avalglucosidase alfa, its investigational enzyme replacement therapy (ERT) for people with Pompe disease. According to a press release, a regulatory decision is expected in the second half of 2021, and if…

Fertility in women is not affected by late-onset Pompe disease (LOPD), but pregnancy may initiate or worsen symptoms, a small study found.  The study, “Pregnancy Outcomes in Late Onset Pompe Disease,” was published in the journal…

While late-onset Pompe disease can affect your ability to perform daily tasks, creative problem-solving skills and support can help you live independently for as long as possible. Here is some information that may help you. About late-onset Pompe disease Pompe disease is a rare genetic disease characterized…

Late-onset Pompe disease can occur during a child’s largest phase of growth and development. This can have a major impact on how well the body develops, including the occurrence of scoliosis. What is scoliosis? Scoliosis is a sideways curve in the spine, and is often related to neuromuscular…

Spark Therapeutics has resumed enrollment into two international clinical studies testing SPK-3006, the company’s investigational gene therapy for Pompe disease. Recruitment of adults with late-onset Pompe disease (LOPD) into both studies — the seroprevalence SPK-GAA-100 study (NCT03893240) and the Phase 1/2 RESOLUTE trial…

Amicus Therapeutics remains on track to announce top-line data from PROPEL — a Phase 3 trial of AT-GAA for the treatment of late-onset Pompe disease — in the first half of 2021, and plans to seek regulatory clearance to start clinical studies of a gene therapy for…

Combining exercise training with a high-protein diet improves exercise tolerance, pulmonary function, and quality of life among late-onset Pompe disease (LOPD) patients taking Lumizyme (alglucosidase alfa) for years, a small study found. The study, “Exercise training alone or in combination with high-protein diet in patients…

A Phase 1/2 clinical trial is recruiting adults with late-onset Pompe disease to test Audentes Therapeutics‘ investigational gene therapy AT845, the company has announced. The trial (NCT04174105) will be conducted at clinical sites in the U.S., Germany, and the U.K. It aims to recruit up to…

Pompe disease is a rare disease in which glycogen, a complex sugar molecule, builds up in cells and tissues. This buildup interferes with function, especially in muscles. Changes to diet and feeding methods may help alleviate or slow the progression of some disease symptoms. One dietary…

Avalglucosidase alfa, an investigational next-generation enzyme replacement therapy (ERT), leads to clinically meaningful improvements in respiratory muscle strength and mobility in children and adults with late-onset Pompe disease (LOPD), according to top-line data from a Phase 3 trial. The findings will be the foundation for applications that…