News

Study examines gene activity changes in LOPD

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,…

Desensitization regimen can help manage anaphylaxis to Myozyme

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, next-gen ERT for Pompe, now available through NHS

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 —…

Pompe Mouse Model Targets Southern Han Chinese Ancestry

Researchers created and characterized an infantile-onset Pompe disease (IOPD) mouse model that carried a genetic defect commonly found among people with Southern Han Chinese ancestry, a study reported. The model recapitulated most of the signs and symptoms of IOPD and, according to researchers, is ideal to evaluate potential IOPD…

Medicine May Open ATA845 Trials to People With AAV Antibodies

Astellas Gene Therapies has obtained the rights to develop medication that could help reduce preexisting immune responses against the viral carrier used in ATA845, its investigational gene therapy for adults with late-onset Pompe disease (LOPD). Called IdeXork, or Xork, the investigational medication was developed by Selecta Biosciences,…