Throughout 2025, the team at Pompe Disease News has brought readers updates on emerging treatments, scientific advances, and clinical…
Articles by Patricia Inácio, PhD
A 1-year-old boy with infantile-onset Pompe disease (IOPD) showed improved muscle strength and signs of less heart inflammation four…
In patients with Pompe disease, treatment with Myozyme (alglucosidase alfa) is most often administered in outpatient settings and is…
While the activity of the acid alpha-glucosidase (GAA) enzyme and glycogen buildup are hallmarks of Pompe disease,…
A man with late-onset Pompe disease (LOPD) was initially misdiagnosed with an inflammatory muscle disease called polymyositis, according to…
The strength of hip muscles and gait wherein only one leg is in contact with the ground were identified as…
A new gene therapy for Pompe disease showed promise in a mouse model, researchers say, fully clearing in muscles…
Regulatory approval in the European Union for miglustat, a component of the two-part investigational therapy AT-GAA for adults with late-onset…
“Every Move Counts” on this year’s International Pompe Day, which is held every year to raise awareness and increase understanding…
No anesthesia complications were reported and surgery was successful for a man with late-onset Pompe disease and airway problems who…