Disease severity varied widely among adults with late-onset Pompe disease who underwent screening to assess their eligibility to participate in a gene therapy clinical trial, a study reports. Elevated levels of antibodies against the viral delivery vector were one of the exclusion criteria, suggesting that future studies may use…
late-onset Pompe disease
Pombiliti (cipaglucosidase alfa) plus Opfolda (miglustat), a two-part therapy formerly known as AT-GAA, has been approved by the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) as a treatment for adults with late-onset Pompe disease (LOPD). “The MHRA approvals for Pombiliti and Opfolda are a major step forward for adults…
A newborn screening program for Pompe disease in Minnesota was found effective for getting babies with infantile-onset disease started on early treatment, but challenges remain for those with late-onset disease (LOPD). “The management of LOPD cases including the establishment of standardized follow up guidelines … and a better…
The enzyme stabilizer Opfolda (miglustat), one part of the two-component therapy formerly known as AT-GAA (cipaglucosidase alfa/miglustat), has been approved by the European Commission to treat adults with late-onset Pompe disease (LOPD). The news comes a few months after the other part of AT-GAA, the enzyme replacement Pombiliti…
Whenever it was time to play dodgeball in my elementary school years, I was always picked last. Growing up, I wasn’t physically gifted. I couldn’t run, and I moved slowly. For a kid, my balance was uncharacteristically off. Despite being raised in Southern California, I was unable to surf,…
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…
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…
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. …
Regulatory authorities in the U.S. and the U.K. are expected to decide in the third quarter of this year whether or not to approve AT-GAA as a treatment for late-onset Pompe disease, according to its developer, Amicus Therapeutics. The U.S. Food and Drug Administration (FDA) had previously…
Late-onset Pompe disease (LOPD) patients exhibit significant gene activity changes in their skeletal muscles compared with healthy people, some of which were normalized after six months of Nexviazyme (avalglucosidase alfa) treatment, a study found. Pathways involved in lysosome function, energy metabolism, and inflammation appeared to be most altered,…