Nearly 1 in 4 adults with late-onset Pompe disease develop high levels of antibodies against enzyme replacement therapy…
Articles by Margarida Maia, PhD
While it is not very common, adults with late-onset Pompe disease (LOPD) may go undiagnosed or misdiagnosed, even if…
Three new mutations in the GAA gene were linked with heart disease and severe breathing problems in infants with…
A diaphragm pacer may help people with Pompe disease who can’t breathe on their own rely less on a…
Starting people with late-onset Pompe disease on enzyme replacement therapy (ERT) at any age can benefit their bone…
Nexviadyme, (avalglucosidase alfa), Sanofi’s next-generation enzyme replacement therapy for Pompe disease, will now be available in the U.K.
FORTIS, a Phase 1/2 clinical trial of an experimental gene therapy called AT845, is due to restart in adults…
The U.S. Food and Drug Administration (FDA) has delayed a decision on whether to approve cipaglucosidase alfa, the biologic component…
A number of enzymes involved in synthesizing glycogen – the molecule that accumulates to toxic levels in Pompe disease…
A toddler girl with infantile-onset Pompe disease is able to walk independently and lives with a healthy heart after…