Two girls in Colombia with early-onset Pompe disease saw their symptoms improve after methotrexate treatment reduced the levels of antibodies…
Articles by Margarida Maia, PhD
Researchers in the U.S. used a gene-editing tool in the lab to correct mutations known to cause infantile-onset Pompe…
Early treatment with at least two cycles of bortezomib may help lower the levels of antibodies that can make…
Sanofi’s Nexviazyme (avalglucosidase alfa), an enzyme replacement therapy approved for Pompe disease, doesn’t appear to be linked…
A gene therapy designed to travel into muscles resulted in widespread production of the enzyme that’s missing or faulty in…
An easy survey of 20 questions agrees well with other tools used in clinical practice to measure physical function in…
Amicus Therapeutics has been honored with the WORLDSymposium 2024 New Treatment Award for Pombiliti + Opfolda (cipaglucosidase alfa/miglustat),…
Despite being on enzyme replacement therapy (ERT) for a long time, some children with infantile-onset Pompe…
Staying physically active according to the World Health Organization (WHO) guidelines and engaging in a personalized exercise program can significantly…
Nearly 1 in 4 adults with late-onset Pompe disease develop high levels of antibodies against enzyme replacement therapy…