|Videos|February 28, 2022
The Benefits of a New Therapy for Pompe Disease: Tahseen Mozaffar, MD, FAAN
Author(s)Tahseen Mozaffar, MD, FAAN
On Rare Disease Day, the director of the Division of Neuromuscular Diseases at UC Irvine commented on the potential of AT845 for this patient population. [WATCH TIME: 3 minutes]
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WATCH TIME: 3 minutes
“The biggest advantage here would be that, rather than being dependent on an exogenously delivered enzyme that has to be given every 2 weeks with a significant amount of infusion-related reaction, plus a time commitment—because the infusion takes about 4 hours—you would be in a situation where your body is producing its own enzyme, either in the liver or in the muscle.”
Pompe disease is a rare neuromuscular disorder affecting 1 person in every 40,000, with approximately 1 out of every 28,000 babies in the US born with the condition. Currently, the only available treatment is enzyme replacement therapy (ERT), which has risk of infusion-related reaction and requires biweekly administration.
Tahseen Mozaffar, MD, FAAN, director, UC Irvine-MDA ALS and Neuromuscular Center, director, Division of Neuromuscular Diseases, Neurology School of Medicine, and professor of neurology, pathology, and orthopaedic surgery, University of California, Irvine, spoke with NeurologyLive® on this data, emphasizing the benefits it may offer patients with Pompe disease, with investigators anticipating the efficacy of AT845 to be higher than that of ERT. Mozaffar further discussed challenges and areas for future research in Pompe disease, noting the potential mRNA approach, which was used by Pfizer/BioNTech and Moderna for their respective COVID-19 vaccines.
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