The benefits seen with Nexviazyme (avalglucosidase alfa) treatment in three people in Japan with Pompe disease matched those found among the overall patient population of two global clinical trials, a new case series analysis by researchers in that country has found. This trio of Japanese patients — two…
treatment
In this column, I’m introducing what I call “The Pompe Community Diaries,” a series that will follow the journeys, musings, and challenges of the fellow Pompe disease patient community. I’ll collaborate with community members to share their voices and day-to-day experiences over time. It’s a space meant to foster…
Last year, the team at Pompe Disease News brought our readers news on the most recent scientific advances, treatment progresses, and clinical trials for Pompe disease. Here are the 10 most read stories we published on Pompe disease during 2024. No. 10 – Switch from Lumizyme to Nexviazyme…
Switching from Lumizyme (alglucosidase alfa) to Pombiliti + Opfolda (a combination of cipaglucosidase alfa and miglustat) may help patients with late-onset Pompe disease move around more easily, suggesting a shift toward better quality of life, according to an exploratory analysis of Phase 3 data. The Phase…
It’s no secret that the holiday season is a busy time for everyone, but imagine having to incorporate a 6.5-hour infusion therapy into the mix of it all. That’s the case for our family because my 6-year-old son, Cayden, has infantile-onset Pompe disease. While planning everything out can be…
Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) may offer significant advantages over earlier forms of enzyme replacement therapy (ERT) for Pompe disease, according to an analysis of preclinical and clinical data. Researchers reviewed all available data about the combination therapy’s mechanism and therapeutic effects and determined that it can overcome challenges…
My 6-year-old son, Cayden, has been seeing an ear, nose, and throat (ENT) doctor since he was about 2 years old. His metabolic disease specialist, who manages everything related to Cayden’s infantile-onset Pompe disease, originally referred him to the ENT department because this type of Pompe is known to…
Aro Biotherapeutics is seeking adults with late-onset Pompe disease (LOPD) to participate in a Phase 1b trial testing ABX1100, the company’s experimental substrate reduction treatment, at a site in Canada. “We anticipate dosing the first patient with Pompe disease in the Phase 1b study before the end of 2024,”…
Myozyme (alglucosidase alfa), sold as Lumizyme in the U.S., eased breathing but did not improve motor function of the arms and legs in an Iranian woman with late-onset Pompe disease who went undiagnosed for many years, according to a case report. The researchers say this case highlights the…
Combination therapy Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) may help people with late-onset Pompe disease (LOPD) walk farther and breathe better compared with Nexviazyme (avalglucosidase alfa), particularly if they’ve been on enzyme replacement therapy for longer, according to a meta-analysis of clinical trials and real-world data. The study, “…
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