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Dec 4, 2017

Show description/summary:1) Long-term benefit of enzyme-replacement therapy in Pompe disease: A 5-year prospective study2) What’s Trending: ACTH or prednisolone for infantile ataxiaThis podcast begins and closes with Dr. Robert Gross, Editor-in-Chief, briefly discussing highlighted articles from the December 5, 2017 issue of Neurology. In the first segment, Dr. Kelly Gwathmey talks with Dr. Nadine van der Beek about her paper on enzyme-replacement therapy in Pompe disease. In the second part of the podcast, Dr. Ted Burns focuses his interview with Dr. John Mytinger on the topic of ACTH or prednisolone for infantile ataxia. DISCLOSURES: Dr. Gwathmey reports no disclosures.Dr. van der Beek has received speaker honoraria from Sanofi-Genzyme (paid to to Erasmus MC for further research purposes); and has received research support from Sanofi-Genzyme, Colciencias, ZonMW - the Netherlands Organization for Health Research and Development, the Prinses Beatrix Spierfonds, SSWO – Sophia Children’s Hospital Foundation, and TKI-Health Holland.Dr. Burns is the deputy section editor of the Neurology® podcast; has served on scientific advisory boards for Argenx, UCB, and CSL Behring; has received travel funding/speaker honoraria from Argenx and Alexion; and has received support for consulting activities from UCB Pharma and CSL Behring.Dr. Mytinger reports no disclosures.