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Featured Blog Posts – April 2010 Archive (4)

Staff Immune Response to Exon Skipping

Responding to concern within the community regarding the potential for an immune response to exon skipping in the recent Quest Magazine article from the MDA, Annemieke Aartsma (Leiden University Medical Center) formulated the answer for families as follows:

First of all, the exon skipping trials are still ongoing, so unfortunately I cannot give you a definitive answer about a possible immune response to dystrophin after exon skipping. However, I… Continue

Added by Pat Furlong on April 20, 2010 at 1:20pm — 5 Comments

Staff Week in Review: 4/11-4/15

I spent the better part of the week in Washington, DC with a full agenda. On Monday, I met with Peter Saltonstall (president of NORD). One of NORD’s major interests is the FDA. You are probably familiar with our advocacy agenda, this year concentrating on several areas relevant to clinical trials – one very specifically directed toward FDA. One of our requests focused on securing additional resources for the FDA, specifically more expertise and…

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Added by Pat Furlong on April 19, 2010 at 11:58am — 6 Comments

Live Report from the American Academy of Neurology

Sharon Hesterlee, Ph.D.

Senior Director Research and Advocacy

Parent Project Muscular Dystrophy…

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Added by Sharon Hesterlee on April 15, 2010 at 5:30pm — 14 Comments

Staff Albania

During the Connect Conference last year, I was invited to speak in Albania. It is hard to say no to invitations during the Conference because the need is so clear and the impact of information so evident. So, I said yes…knowing it would be sometime in the future and maybe, depending on the circumstances, might not happen. In January, Mira contacted me reminding me of our conversation and the scheduled date, March 27. In January, the end of March seemed far away.

I met Mira some years… Continue

Added by Pat Furlong on April 6, 2010 at 1:04pm — 2 Comments