The U.S. Food and Drug Administration (FDA) has cleared Aro Biotherapeutics to conduct a Phase 1 clinical trial to…
Articles by Andrea Lobo, PhD
Young patients with late-onset Pompe disease (LOPD) who do not yet show disease symptoms should be regularly monitored for…
Last year, the team at Pompe Disease News brought our readers news on the most recent scientific advances, treatment…
Three genes related to the regulation of immune responses — GPNMB, CALML6, and TRIM7 — show abnormal activity in immune…
Dyne Therapeutics has unveiled promising preclinical data for its enzyme replacement therapy (ERT) FORCE-GAA — part of the company’s…
Health-related quality of life for children with Pompe disease in the Netherlands is generally similar to healthy peers when…
An immune system modulatory regimen effectively reduced levels of antibodies against Myozyme (alglucosidase alfa), an enzyme replacement therapy (ERT)…
Switching to Nexviadyme (avalglucosidase alfa) from Myozyme (alglucosidase alfa) can help to halt the worsening of motor function…
The COVID-19 pandemic affected the treatment, as well as the physical and mental health, of people with Pompe disease,…
Pompe disease is less prevalent than expected in Spain, at around 3.1 cases per million inhabitants, according to a…