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, “…
Pombiliti plus Opfolda, a combination treatment developed by Amicus Therapeutics for adults with late-onset Pompe disease (LOPD), has been selected as the winner of the 2024 Prix Galien U.K. Award for Best Pharmaceutical Product. This award is given by the Galien Foundation to recognize companies that have helped…
Long-term treatment with Pombiliti plus Opfolda (cipaglucosidase alfa/miglustat) safely improved motor function and stabilized or enhanced lung function in adults with late-onset Pompe disease (LOPD), according to newly published data from two clinical trials. Pompe disease is marked by a deficiency in an enzyme called acid alpha-glucosidase…
Throughout 2023, Pompe Disease News brought you coverage of the latest developments in research into Pompe disease and clinical trials of potential new treatments. Here is a list of the 10 most-read stories we published across that year. We hope to continue being a source of information…
Pombiliti (cipaglucosidase alfa) plus Opfolda (miglustat), a combination therapy formerly known as AT-GAA, has been approved by the U.S. Food and Drug Administration (FDA) to treat certain adults with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT). Its developer, Amicus…
Get regular updates to your inbox.