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A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease.
Byrne BJ, Geberhiwot T, Barshop BA, Barohn R, Hughes D, Bratkovic D, Desnuelle C, Laforet P, Mengel E, Roberts M, Haroldsen P, Reilley K, Jayaram K, Yang K, Walsh L; POM-001/002 Investigators. Byrne BJ, et al. Orphanet J Rare Dis. 2017 Aug 24;12(1):144. doi: 10.1186/s13023-017-0693-2. Orphanet J Rare Dis. 2017. PMID: 28838325 Free PMC article. Clinical Trial.
Screening for late-onset Pompe disease in Poland.
Jastrzębska A, Potulska-Chromik A, Łusakowska A, Jastrzębski M, Lipowska M, Kierdaszuk B, Kamińska A, Kostera-Pruszczyk A. Jastrzębska A, et al. Acta Neurol Scand. 2019 Oct;140(4):239-243. doi: 10.1111/ane.13133. Epub 2019 Jun 18. Acta Neurol Scand. 2019. PMID: 31125121