The enzyme stabilizer Opfolda (miglustat), one part of the two-component therapy formerly known as AT-GAA (cipaglucosidase alfa/miglustat), has been…
Articles by Marisa Wexler, MS
The switch to at-home infusions of enzyme replacement therapy (ERT) — made necessary due to the COVID-19 pandemic —…
People with late-onset Pompe disease (LOPD) rarely have genetic variations in genes that are important for making glycogen in…
A surgical technique called conjoint fascial sheath (CFS) suspension may help relieve drooping eyelids in children with infantile-onset Pompe…
After two years being treated with AT-GAA (cipaglucosidase alfa/miglustat), adults with …
Nearly three years of treatment with Nexviazyme (avalglucosidase alfa) led to long-term improvements…
Three siblings with Pompe disease caused by the same mutations were described in a recent report. Despite all having…
Regulatory authorities in the U.S. and the U.K. are expected to decide in the third quarter of this year whether…
Researchers in Taiwan have developed new molecules that can stabilize the acid alpha-glucosidase (GAA) enzyme, a strategy they say might…
MRI scans of people with classic infantile Pompe disease showed a change in the microscopic structure of certain brain…