News

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…

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. …

Three siblings with Pompe disease caused by the same mutations were described in a recent report. Despite all having the same disease-causing mutations, the clinical presentation of the three children differed dramatically — one began showing symptoms in infancy, while another had not shown clinical abnormalities at the age…

Single and multiple ascending doses of MZE001, Maze Therapeutics‘ experimental oral substrate reduction treatment for Pompe disease, were well tolerated and reduced glycogen levels in healthy volunteers in a Phase 1 trial, data show. These findings support a Phase 2 trial of MZE001 in people with Pompe disease,…

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,…

Anaphylaxis, a severe allergic reaction, occurred in nearly one-third of children with Pompe disease treated with the enzyme replacement therapy Myozyme (alglucosidase alfa) in a small study. However, rapid desensitization regimens — using small doses initially and then increasing the doses — safely re-established tolerance to Myozyme in…

Nexviadyme, (avalglucosidase alfa), Sanofi’s next-generation enzyme replacement therapy for Pompe disease, will now be available in the U.K. through the National Health Service (NHS). That’s according to a review from the National Institute for Health and Care Excellence that deemed Nexviadyme as an option for treating people —…