At the annual congress of the World Muscle Society (WMS) in Geneva, September 9th-12th, Nadine van der Beek and Juna de Vries presented the results of Enzyme Replacement Therapy (ERT) in 73 Dutch adult Pompe patients on muscle strength and pulmonary function. The two young research physicians work at the department of Neurology and the Center for Lysosomal and Metabolic Diseases of Erasmus MC, Rotterdam, The Netherlands. Because alglucosidase alfa (Myozyme®) is an orphan drug, the center serves as a national referral center for all Pompe patients in The Netherlands.
Currently 93 adult patients are followed at the Erasmus MC, of whom 73 patients receive ERT with alglucosidase alfa (dose of 20 mg/kg every 2 weeks). Preliminary results show that ERT has a positive effect on muscle strength and seems to stabilize pulmonary function. Effect of treatment however varies among individual patients. Further subgroup analyses and longer follow-up duration are performed to identify possible prognostic factors. This knowledge will be used to optimize treatment in the future and set-up treatment guidelines.
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