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FDA, Medical Product Regulation, & Advocate Involvement Webinar

CureDuchenne — Mon, 12 Jul 2010 16:00:08 +0000

FDA, Medical Product Regulation, and Advocate Involvement

How does the FDA make its decisions? Who makes the rules for drug development? What special accommodations are made for rare diseases?

Cure Duchenne and Parent Project Muscular Dystrophy are happy to bring you this two-part live Webinar series featuring Dr. David Banks of the FDA Office of Special Health Issues. Dr. Banks will walk us through the general steps required for approving a drug with special emphasis on the implications of the Orphan Drug Act, individual INDS and expanded access programs. This series is meant to be interactive-participants can either type in questions that will be displayed during the discussion or submit questions in advance. These talks will attempt to provide some insights into the FDA’s mission-what are its constraints, and what are its goals?

Dr. Banks will not be able to address questions about specific therapeutics that are undergoing regulatory review right now, but can talk about general principles and how they might be applied. The topics in the two series will be different.

To submit advance questions please respond to: Debra Miller – debra(at)cureduchenne.org

The first webinar will be this week on July 15th, 3pm Eastern (Noon Pacific)

To Register, Click Here

CureDuchenne Founder Addresses Medical Students at UCI with Updates on Duchenne Muscular Dystrophy Debra Miller, founder of CureDuchenne was invited...

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2010 Climb to Cure Duchenne Raises over $250,000 for DMD Research

CureDuchenne — Tue, 22 Jun 2010 23:20:59 +0000

Take a good, long look at the above photo. What do you see? You might see a man on a mountain top. You might see the snow. You might see the clouds in the background.

However, if you look a bit deeper, you’ll realize there is a story behind this picture. Paul Miller, the man seen standing triumphant with ice ax in hand, is the father of a boy with Duchenne muscular dystrophy. This picture was taken at the summit of Mt. Shasta in Northern California at 14,162 feet in below freezing temperatures.

This picture is merely a symbol of the dedicated parents and DMD advocates from around the country that participated in this year’s 2010 Climb to Cure Duchenne: Pick Your Peak.

The climb consisted of literally hundreds of people all over the country who chose to assemble a team, pick a peak, and climb on June 19th to raise awareness and money for a cure for Duchenne muscular dystrophy. It was an amazing event for everyone involved.

The climb was successful in two primary and extremely important ways. First, more than ever, people from around the country were introduced to Duchenne muscular dystrophy and learned about the cause. Because of this event, there are many people who have signed on to help find a cure.

Second, this event raised more than $250,000 that will go towards research! In fact, Cure Duchenne will allocate the funds to Dr. Chamberlain, of the University of Washington, and Dr. Stephen Tapscott, of the Fred Hutchinson Cancer Research Center, who have identified a promising gene therapy that could lead to a treatment of all boys afflicted with DMD. This is very big news!

Finding a cure to something as big a DMD is going to require massive action. It’s going to require getting out there and making a difference. It’s going to require doing for our boys what they can’t do for themselves.

Cure Duchenne is extremely grateful to all those who took action and participated in this year’s Climb to Cure Duchenne.

So when you look at a photo like the one of Paul Miller above, recognize that the struggle he underwent is nothing compared to the struggle our boys go through on a daily basis. For all those who want to see a cure found, join us in taking massive action and help Cure Duchenne!

Announcing the 2010 Climb to Cure Duchenne: Pick Your Peak The 2009 Climb to Cure Duchenne was very successful in every...
2010 Climb to Cure Duchenne: Pick Your Peak! Announcing this year’s Climb to Cure Duchenne: Pick Your Peak!...
2010 Climb to Cure Duchenne: A Message from Cure Duchenne Founder, Debra Miller This week CureDuchenne was very happy to announce our first...

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Dealing for Duchenne Celebrity Poker Tournament Raises Over $200,000

CureDuchenne — Mon, 21 Jun 2010 19:00:03 +0000

This year’s “Dealing for Duchenne” Celebrity Poker Tournament was a smashing success! We had record numbers with over 450 people in attendance. The night’s event helped to raise more than $200,000 that will go towards UCLA DMD Research & Clinic. This is very big news for everyone affected by DMD here on the West coast and Southwest region.

Special thanks go to Amy & Chris Martin who chaired the event. Amy & Chris are experts at putting together an amazing team and making a perfect evening happen. Thank you to everyone involved!

If you would like to donate to the UCLA DMD Clinic, please click here:

Enjoy some photos from the evening:

Dealing for Duchenne Los Angeles: Celebrity Poker Tournament with Co-Hosts Anne Heche and James Tupper On June 12th, CureDuchenne is pleased to have Anne Heche...
UCLA Hopes for Duchenne Muscular Dystrophy Clinic At CureDuchenne, we’re very pleased to announce the plans for...
CureDuchenne Grants $200,000 to UCLA Duchenne Muscular Dystrophy Research & Clinic On November 10th, 2009 Debra Miller, Founder and President of...

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