late-onset Pompe disease

Regulatory approval in the European Union for miglustat, a component of the two-part investigational therapy AT-GAA for adults with late-onset Pompe disease (LOPD), has been recommended by a branch of the European Medicines Agency (EMA), the body responsible for monitoring drug therapies in the EU. A decision on whether or…

Last week, my 5-year-old son, Cayden, had a checkup with his metabolic doctors during a clinic day for his Pompe disease. This visit was mostly typical of those events and went about as expected. I was thankful for the good report from his doctors. Further, the physical therapist said…

A long time ago, in a galaxy far, far away, I was celebrating 36 years on this planet. I’d just returned from the mountains in my landspeeder when I received a phone call from my sister. My dad, who was five months into his second round of…

No anesthesia complications were reported and surgery was successful for a man with late-onset Pompe disease and airway problems who broke a bone in his upper arm, according to a case report from Spain. The man, 47, underwent surgery for the broken left arm under a combined anesthesia approach after…

Back in the summer of 2018, when I started experiencing some serious symptoms, I wondered if I had ALS or another similar disorder. I noticed I wasn’t able to stand up from a seated position. If I was sitting on the couch, I’d have to extend my arms…

After two years being treated with AT-GAA (cipaglucosidase alfa/miglustat), adults with late-onset Pompe disease (LOPD) maintained their improvements in walking ability and had stabilized breathing function, as well as a reduction in the levels of disease biomarkers, according to…

Pombiliti (cipaglucosidase alfa), a component of AT-GAA, a two-part investigational therapy, has been approved to treat adults with late-onset Pompe disease (LOPD) in the European Union. The European Commission (EC) ruling comes on the heels of a positive opinion issued by the Committee for Medicinal Products for Human…

Nearly three years of treatment with Nexviazyme (avalglucosidase alfa) led to long-term improvements in walking ability and lung function for people with late-onset Pompe disease (LOPD), according to new data from the Phase 3 clinical trial COMET. …

Today, March 17, is the day to celebrate all things green and Irish. Look for a four-leaf clover and have some Lucky Charms for breakfast because they’re “magically delicious.” Drink some green beer and have corned beef and cabbage for dinner. Search for the pot of gold at the end…

Every other week, my son Cayden, who’s almost 5, receives an enzyme replacement infusion as part of his treatment for infantile-onset Pompe disease. The infusion typically takes about six hours, and although we’re used to the routine, it does make for a very long day. Cayden has…