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Past AMDA Webinars

Design And Performance Of Clinical Trials: An Overview
Title: Design and Performance of Clinical Trials: An Overview
Date: Monday, October 20, 2014
Speaker:
Anne Zajicek, MD, PharmD, FAAP, Chief, Obstetric and Pediatric, Pharmacology and Therapeutics Branch, National Institute of Child Health and Human Development (NICHD)

The Contribution Of The IPA Survey To Our Understanding Of The Natural Course Of Pompe Disease
Title: The contribution of the IPA survey to our understanding of the natural course of Pompe disease and effects of enzyme therapy: Ten years of the IPA survey
Date: Thursday, February 27, 2014
Speaker:
J.C. van der Meijden, Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, the Netherlands
Webinar Overview:
For more than ten years Pompe patients, from all over the world, participate in the International Pompe Association / Erasmus MC Pompe survey on a yearly basis. During this period we have gathered a vast amount of information on the natural course of Pompe disease and the effects of enzyme replacement therapy. During this webinar we will discuss what we have learned about Pompe disease by working with the Survey. Furthermore, we will discuss the importance of the survey and goals for the future. At the last part of the webinar there is an opportunity for participants to ask questions to the researchers.

Needle Core Muscle Biopsy
Title: Needle Core Muscle Biopsy
Date: Wednesday, March 7, 2012
Speaker:
Mark Tarnopolsky, MD, PhD, FRCP (C)
Webinar Overview:
Traditionally most Neuromuscular Clinics have used an open muscle biopsy for pathological diagnosis. Many Neuromuscular Specialists and Neuropathologists have avoided the use of a needle biopsy technique due to small sample size. The UCH needle has a port on the side to apply suction and this definitely increases the yield several fold. There are several strategies to apply suction to the Bergstrom needle and we will describe custom modifications that we have found to yield excellent sample size. The advantages of the needle biopsy technique are; much smaller scar, very high safety profile; adequate tissue size; ability to perform the biopsy within ten minutes in the clinic; and the ability to sample several different muscles that is a function of the EMG and/or clinical examination. This presentation will go over the needle muscle biopsy technique using either the UCH or Bergstrom muscle biopsy needle for research and patient diagnosis.