Using self-reported outcomes by adults with Pompe disease, researchers were able to estimate the minimal clinically important difference or MCID…
Articles by Steve Bryson, PhD
Long-term treatment with Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) safely improved motor function and stabilized or enhanced lung function in…
Switching from Lumizyme (alglucosidase alfa) to Nexviazyme (avalglucosidase alfa) was shown to improve breathing and motor function for…
Children with classic infantile-onset Pompe disease (IOPD), who were first treated as infants with Myozyme (alglucosidase alfa) — sold…
Disease severity varied widely among adults with late-onset Pompe disease who underwent screening to assess their eligibility to participate…
Hypersensitive reactions to infusions of enzyme replacement therapies (ERT) to treat late-onset Pompe disease (LOPD) were primarily mild and…
Motor problems in people with Pompe disease may be explained by damage to muscle spindles, which are sensory receptors…
Pombiliti (cipaglucosidase alfa), a component of AT-GAA, a two-part investigational therapy, has been approved to treat adults with…
Anaphylaxis, a severe allergic reaction, occurred in nearly one-third of children with Pompe disease treated with the enzyme replacement…
Researchers created and characterized an infantile-onset Pompe disease (IOPD) mouse model that carried a genetic defect commonly found among…