Featured Blog Posts – November 2009 Archive (7)

A moment for thanks

We spend most of our lives in running shoes. This past year in particular, there were added worries, and it felt like we had to run stronger and faster, often without taking a moment to catch our breath.

On Thanksgiving, it is important to loosen those laces, put the shoes in a corner, and take a moment to bow our heads and give thanks.

I am thankful…

•…for amazing advances in research. Our End Duchenne Grant Award Program invested almost $1 million in research,… Continue

Added by Pat Furlong on November 25, 2009 at 10:11am — 19 Comments

HOPE – finding the Therapeutic Dose

It’s not easy. The word Duchenne enters our lives. We search the internet, seek out expertise, connect with researchers, clinicians, industry, other families, set up our internet tools (Google alerts, RSS feeds, list-serves, pubmed search criteria, industry alerts, etc) and live each day hoping to see our ‘in-box’ overflowing with news, with HOPE.

On any given day, you reassure yourself that your son is young, may have a certain mutation, may have a certain degree of muscle function,… Continue

Added by Pat Furlong on November 23, 2009 at 3:10pm — 7 Comments

Notes from Treat-NMD Conference

Phase I/IIa study on Antisense Compound PRO051 in patients with Duchenne (van Deutekom)

Eight years in the drug development of AON. PRO051 is a 2’o-Me-PS RNA antisense oligonucleotide introducing exon 51 skipping. It demonstrated proof of concept with up to 95% dystrophin fibers in treated muscle area in Duchenne patients. 6 month safety data on PRO051 was available in monkeys and longer term studies in progress. Extensive safety data available for this class of compounds. 12… Continue

Added by Pat Furlong on November 18, 2009 at 1:34pm — 10 Comments

News from Brussels

Currently in Brussels for the Treat NMD Advisory Committee for Therapeutics Conference.

How many times have we all received Google Alerts suggesting a certain drug "is potentially promising," “improves function in the mdx mouse,” or ‘halts degeneration in the mdx”? And then, silence.

And how many of us have discussed the need for a certain drug to go to trial or how many parents/families have tried to persuade or were successful at persuading their physician to write the… Continue

Added by Pat Furlong on November 17, 2009 at 4:28pm — 6 Comments

MD Breakthrough - What to Make of It

Whenever I hear the words "MD Breakthrough', my heart skips a beat. Is it really? Often, it is in reference to a study in the mdx mouse. We can cure mice. But people are not mice... just ask Victor Dubowitz. And when the 'breakthrough' talks about a young man with Duchenne, it feels like we are all standing at Attention!

Over the last couple of days, many of you have seen a news story that recently aired about a young man named Ryan. I wanted to share with you my thoughts… Continue

Added by Pat Furlong on November 10, 2009 at 4:28pm — 46 Comments

Joshua Arndt

Joshua died last Monday. There are no words to soften the blow. Losing one of our sons hurts all of us. The world is just not the same, never will be. We can ask ‘why’ and say all of the expected things, but it does not sync. Losing Joshua is unacceptable.

Our heart is broken again. He was one of us, he is our son. His parents are dear friends, colleagues, fighters in this difficult battle to EndDuchenne.

Our sons teach us everyday of their lives. They teach us how to… Continue

Added by Pat Furlong on November 9, 2009 at 10:30am — 7 Comments

Jenny's Wedding

When you have children, the concept of marriage/partner/weddings comes to mind now and again. I scrutinize everyone – ask the girls! I try my best not to state my opinion, but it’s probably obvious. In the past, when relationships fail, I have been known to be a bit vindictive…ok, even thinking about voodoo – shredding one young man’s photograph and later hearing a cat vomited on his bed. It made me pretty happy! As you might imagine, Jenny and Michelle may at times be reluctant to make… Continue

Added by Pat Furlong on November 4, 2009 at 10:43am — 9 Comments

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