Gene therapies — after a tragedy kicked them back to the lab two decades ago — are beginning to come into their own, making possible the progress in gene-targeted and combination treatments for neuromuscular diseases once thought unimaginable. But many challenges remain — from the likelihood of no “one and…
Dosing
Amicus Therapeutics has dosed the first patient in the Phase 3 PROPEL clinical trial that aims to compare investigational therapy AT-GAA (ATB200/AT221) to standard treatment in patients with late-onset Pompe disease. The trial (NCT03729362) is still recruiting patients. Throughout 2019, approximately 100 participants who have been previously…
Early treatment with a four times higher dose of Myozyme (alglucosidase alpha) was safe and enabled normal motor development and normalization of markers of muscle damage and disease progression in a boy with classic infantile-onset Pompe disease, according to a study. The study, “Early…
Treatment with Lumizyme (alglucosidase alfa) at double the approved dose may help prevent the rapid decline of respiratory and cardiovascular functions in infants with Pompe disease, a case report suggests. Since the U.S. Food and Drug Administration approved it in 2006, Lumizyme has change the course of Pompe…
Recent Posts
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- Wanting to blend in, so the rare care isn’t the first thing that’s seen