For people with late-onset Pompe disease (LOPD) who are diagnosed before developing symptoms, treatment can be delayed until signs…
Articles by Marisa Wexler, MS
Screening people with unexplained muscle weakness or elevated levels of muscle damage markers can help to identify people with…
A two-step process used to screen thousands of people with suspected Pompe disease led to diagnoses of the…
Aro Biotherapeutics is seeking adults with late-onset Pompe disease (LOPD) to participate in a Phase 1b trial testing ABX1100,…
Difficulty swallowing is a common problem for adults with late-onset Pompe disease (LOPD) and can have a sizeable impact…
Switching from Myozyme (alglucosidase alfa) to Nexviadyme (avalglucosidase alfa) led to gains in motor function and laboratory markers…
A group of experts in Europe published updated recommendations for late-onset Pompe disease (LOPD) treatment, including for the first…
Pombiliti plus Opfolda, a combination treatment developed by Amicus Therapeutics for adults with late-onset Pompe disease (LOPD), has…
For most adults with late-onset Pompe disease (LOPD), a fairly significant improvement in walking ability is needed to be…
Most people with classic infantile-onset Pompe disease (IOPD) develop brain abnormalities despite treatment with Myozyme (alglucosidase alfa), and…