Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask)

Event Details

Dystrophin 101: Everything You Always Wanted to Know About the Duchenne Protein (And Were Not Afraid to Ask)

Time: March 3, 2015 from 1pm to 2pm
Location: Online Webinar - Eastern Time Zone
Website or Map: http://www.readytalk.com/
Phone: 866.740.1260 -- Access code: 9449985
Event Type: webinar
Organized By: PPMD
Latest Activity: Mar 3, 2015

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Event Description

March 3, 2015 from 1pm to 2pm ET

This webinar will review the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrophin-glycoprotein complex, characteristics of different forms of dystrophin (both naturally occurring and drug-induced truncated forms), discuss how dystrophin is measured in both preclinical studies and clinical trials, and discuss the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. We will also discuss what is required in order to have dystrophin considered as a surrogate.

Questions

Questions can be emailed to info@parentprojectmd.org by Monday, March 2 at noon.

Speakers:

  • Professor Francesco Muntoni, Chair of Paediatric Neurology and Director of the Dubowitz Neuromuscular Centre, University College of London
  • John Porter, Ph.D., CEO, Parent Project Muscular Dystrophy

To Participate:

  1. Visit ReadyTalk.com and use participant code 9449985. (Be sure to test your computer beforehand)
  2. Audio Dial-In Information:
    • U.S. & Canada: Dial 866.740.1260 and use the Access Code 9449985
    • Outside the U.S. and Canada: Lookup your number

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