Duchenne Muscular Dystrophy Research Fund





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DMDfund
PO Box 17371
Encino, CA 91416
Phone: (818) 692-5500 
Email:

Duchenne Muscular Dystrophy
Research Fund (DMDfund)


About Duchenne Muscular Dystrophy (DMD)
  • Duchenne Muscular Dystrophy (DMD) is the most common lethal genetic disease of children worldwide.
  • It is 100% fatal.
  • DMD is a progressive weakening defect of all the muscles in the body, including the heart, and primarily occurs in boys.
  • There is no cure, no treatment, and no survivors.
  • Approx. 1 in every 2,400 boys worldwide is born with DMD; they die, on average, at about 16 years old.
  • In about 60% of the cases, DMD is inherited from the mother; in about 40% of the cases the disease is the result of spontaneous gene mutation, meaning anyone's son could be born with DMD.
  • DMD is a worldwide tragedy; kids of all colors, all nationalities, all classes, and all ethnic backgrounds die much too young, agonizingly, slowly, and painfully, from DMD. [more]

Message From Kyle Kyllan
DMDfund President

dmdThis is my nephew, Alex.

Alex is a typical 16 year-old boy, high-spirited, imaginative, full of life. But with no future.

Alex has Duchenne Muscular Dystrophy, a relentlessly progressive muscle-wasting disease that will kill him in a few short years. [more]
DMD: A Wake–Up Call
By Dr. Timothy Cripe, MD, PhD

“Your child has cancer” is devastating news, and until today, I thought it was the worst news that a parent could receive.

I now believe that “your child has Duchenne Muscular Dystrophy” needs to sit at the top of the list.

I am a pediatric oncologist and cancer researcher, and unfortunately have had the opportunity to witness the ravages of a cancer diagnosis to both the child and the family. [more]

Alex & cousin Kevin Weisman
("Marshall" on ABC-TV's hit show "Alias")
A Research Success Story!

Drug shows promise for muscular dystrophy, other genetic diseases

The treatment is found to be effective against muscular dystrophy in mice, and holds promise for many other illnesses.

By Thomas H. Maugh II, Los Angeles Times, Apr 28, 2007

[more]


A Research Success Story!
By Thomas H. Maugh II

Drug shows promise for muscular dystrophy, other genetic diseases.

The treatment is found to be effective against muscular dystrophy in mice, and holds promise for many other illnesses.

An experimental drug can effectively cure Duchenne muscular dystrophy in mice by correcting a genetic defect and allowing healthy muscles to develop, researchers reported Sunday in a paper published online by the journal Nature.

Preliminary studies in humans with muscular dystrophy and cystic fibrosis suggest the drug is safe, well-tolerated and potentially effective, according to a team headed by H. Lee Sweeney, chairman of physiology at the University of Pennsylvania School of Medicine.

In addition to Duchenne Muscular Dystrophy — a prevalent, rapidly worsening form of the disorder — the drug has the potential to cure a host of genetic diseases caused by the same mutation, Sweeney said. 

 "There are literally thousands of genetic diseases that could benefit from this approach," he said. "What's unique about this drug is it doesn't just target one mutation that causes disease, but a whole class of mutations."

 The mutations are called premature termination codons, or PTCs. These mutations tell the cell's protein-making machinery to stop synthesis before a protein is completely assembled. In the case of  muscular dystrophy, the mutated gene — which is present in about 15% of MD patients — leads to the production of a truncated form of dystrophin, a crucial protein that helps keep muscle cells intact.

About 10 years ago, Sweeney discovered that the antibiotic gentamicin could override PTCs to allow production of intact proteins like dystrophin.

But it required high concentrations of the drug, which produced severe side effects.

His team subsequently screened more than 800,000 compounds until they found the current candidate, called PTC124. This drug overrides the mutated termination codon, but not the normal stop codon at the end of the gene. It is produced by PTC Therapeutics of South Plainfield, N.J. Sweeney is a member of the company's scientific advisory board.

In the new study, his team administered the drug to so-called mdx mice, which carry the premature termination codon. Within two to eight weeks, the researchers detected full-length dystrophin in the animals. Although it was produced at low levels, it was enough to produce healthy muscles.

Enough dystrophin accumulated in the muscles of the MD mice so that we could no longer find defects in the muscles when we examined them," Sweeney said.

The new findings indicate that "there is now hope that PTC124 … could be more beneficial in the clinic" than gentamicin, German researchers wrote in an accompanying editorial.

PTC Therapeutics is conducting trials in muscular dystrophy patients in the United States and in cystic fibrosis patients in the U.S. and Israel.

Preliminary results reported by the company last year indicated boys receiving the treatment for MD had greater activity and increased endurance.

The cystic fibrosis patients showed decreased sputum volume and thickness, decreased frequency and severity of coughing, and a better sense of wellbeing, the company said.

By Thomas H. Maugh II, Los Angeles Times, Apr 28, 2007

DMDfund helped fund this research. Although it will not help our nephew Alex, it may help 10-15% of the kids with DMD, as well as millions of people with other genetic diseases.

Much more research is desperately needed to help the remaining 85-90% of the DMD kids. Please help fund more research by donating generously to the DMDfund now!


The DMDfund


The Organization
DMDfund is a non-profit 501 (c)(3) organization founded by parents, relatives and friends of kids who have DMD. DMDfund is dedicated to finding a cure for DMD by funding research, so that millions of kids worldwide can live.

The DMDfund is a distinct and independent organization, and is not
affiliated with, nor receives any funding from, any other organization.

Goal: $35 Million Dollars. Partial List of Immediate Needs
$1.5 million for a pilot project for research on treatment of DMD by implanting mesenchymal stem cells, to be performed at Children’s Hospital in Los Angeles.

$4 million to fund 40 new PhD research associates for 2 years. These researchers are desperately needed to continue the fight to find a cure for DMD.

$5 million to establish DMD clinical fellowships at local medical schools, as currently, there are no DMD specialists in the medical community.

Contact Us
Kyle Kyllan, Founder and President • 818-692-5500

Hearts of Gold
David Anders ("Alias")
Billy Burke ("24")
Robert Carradine ("Lizzie McGuire")
Majandra Delfino ("Roswell")
Don Felder (The Eagles)
Devon Gummersall ("The L Word")
LaLaine  ("Lizzie McGuire")
Shannon Lucio ("The O.C.")
Jake Thomas ("Lizzie McGuire")
Jim Turner ("Arli$$")
John Zaring & Bianca Cioffi (Zaring/Cioffi Entertainment)
Chris Pirillo & Ponzi (Lockergnome.com)
Bill Boyd and Amy Striebel (Dick Clark Productions)
Dr. Jeffrey Chamberlain (U of Washington School of Medicine)
Koren Stark & Zach
Don & Yvonne Hill & Joey
Jim Picicci
Tim Conway ("The Carol Burnett Show")
Tom Everett Scott ("Saved")
Greg Bowman
Dave Manship (Carey-It-Off Prod.)
Bruno Racineux (Hexanet.com)


ABC-TV's "Alias"
JJ Abrams
Jennifer Garner
Victor Garber
Michael Vartan
Ron Rifkin
Carl Lumbly
Bradley Cooper
Merrin Dungey
Kevin Weisman


How to Donate
To access the donation form please [click here].



Duchenne Muscular Dystrophy: A Wake-Up Call

Timothy P. Cripe, MD. PhD
Associate Professor of Pediatrics
Division of Hematology/Oncology
Director, Translational Research Trials Office
Cincinnati Children's Hospital Medical Center


"Your child has cancer" is devastating news and until today I thought it was the worst news that a parent could receive. I now believe that "your child has Duchenne Muscular Dystrophy" needs to sit at the top of the list. I am a pediatric oncologist and cancer researcher and unfortunately have had the opportunity to witness the ravages of a cancer diagnosis to both the child and the family. There is no question that the diagnosis of cancer is bad news, but I am proud to say that there has been significant progress in the field. We are at least running a reasonably effective offensive attack against many pediatric malignancies. Unfortunately, the same cannot be said for boys affected by Duchenne Muscular Dystrophy (DMD).

Because my wife is a pediatric cardiologist, our family recently attended the closing banquet of the Parent Project Muscular Dystrophy annual meeting. At the meeting there were ~250 families affected by Duchenne Muscular Dystrophy gathered together to promote research and education in DMD. At our table was an adorable, blond six-year-old boy named Charlie. He had that generic "healthy boy-look," with no outward difference apparent from my two sons, who were seated across the table. However, there was an important difference. Charlie is a ticking time bomb as he was recently diagnosed with DMD. If there are no significant research advances made soon, Charlie will face certain death sometime within the next 10-15 years. And it won't be pretty. It will be a slow, painful, downhill spiral. Charlie will eventually lose the ability to walk followed by the ability to breathe. His heart will slowly lose its ability to function. At this moment in time, there is little that anyone can do about it. Unfortunately, medical science has not orchestrated any effective treatment strategies against the disease that is slowly destroying the muscles in his body.

Thirty years ago, that's the way it used to be with many types of childhood cancer: almost guaranteed death. We can now cure nearly 70% of children with cancer. Forces in the U.S. were mobilized with a 1972 Presidential declaration of war on cancer, and legions of scientists and doctors worldwide have since spent countless years and dollars investigating the biology and treatment of cancer. Childhood cancer afflicts 1 in 300 children younger than 20 years old in the United States (data from the National Cancer Institute SEER Program).

That number includes many readily treatable forms of cancer. Because of hundreds of clinical trials conducted by multiple cooperative groups, only about 1 in 1,000 children die from cancer. As a result of these efforts, hearing that your child has cancer is no longer a death sentence.

The incidence of DMD is 1 in ~3,000 male births. However, in contrast to cancer, DMD currently is uniformly fatal. As a result there is only a 3-fold difference in the number of childhood deaths due to cancer vs. DMD, but DMD research is relatively scarce. A search of the NIH website for open clinical trials (nih.gov; searched 7/18/03) revealed 2,368 for cancer (including 936 that were open to children.) There were only 3 for DMD, a 790-fold difference. The paucity of research may be the reason that there is no cure for this devastating neuromuscular disease. Without research, there is no hope. Shouldn't all people at least been given the chance to hope?

Why the disparity in resource allocation? Why has no war been declared on muscular dystrophy? Whatever the reason, it's time to change. Every person, even a blonde little boy, should at least be able to face tomorrow with hope. Having the opportunity to meet Charlie and his family was a wake-up call.


*Posting of this letter on the DMDfund site is by permission from Dr. Timothy P. Cripe.

DMDfund Notes:

The statistics provided by Dr. Cripe refute the notion that DMD is an “orphan disease.”

In contrast, DMD is the most common fatal genetic disease of children.

The statistics state that there is a 3:1 ratio of children’s deaths from cancer vs DMD. (The ratio is 2.4:1 if the statistic, utilized by DMDfund, of the incidence of DMD is 1 in ~2,400 male births.)

In a perfect world, there would then be a 3:1 difference in the number of cancer clinical trials vs the number of DMD clinical trials. (2.4:1 using the DMDfund numbers.) Instead, the ratio is 790 cancer clinical trials to 1 DMD clinical trial ! (312:1 if only the pediatric trials are considered.)

Conclusion: There is not nearly enough research being done on one of the leading causes of death of children worldwide!



Message from Kyle Kyllan, DMDfund President

Duchenne Muscular Dystrophy

This my nephew, Alex.

Alex is a typical 16 year-old boy; high-spirited, imaginative, full of life. But with no future.

Alex has Duchenne Muscular Dystrophy (DMD), a relentlessly progressive muscle-wasting genetic disease that will kill him in a few short years.

DMD is 100% fatal. There is no cure, no treatment, no survivors. One in every 2,400 boys worldwide is born with DMD.

Alex has been confined to a wheelchair since he was 8.   He can no longer raise his hands to his mouth to feed himself. He has to wear a respirator at night to help him breathe.

The average age of death of boys with DMD is about 16 – Alex’s DMD is progressing so rapidly that he probably won’t make it to that age.

The only hope for Alex, and millions of other boys with DMD, is that a cure, or even a treatment to delay the progression of the DMD, will be found soon.

Alex needs your help. He’d like to be a typical 12 year-old boy, with many years of life ahead to enjoy.

He would like a future.

Research is his only hope. And research costs money. A great deal of money.

Won’t you please help by donating generously to the DMDfund so we can fund that desperately needed research?


Kyle Kyllan
President of DMDfund


"Darius Goes West: The Roll of His Life"
"...a wonderful, heartwarming (and very funny), award-winning documentary..."

Synopsis: A crew of college-age students takes Darius Weems, 19, who has Duchenne Muscular Dystrophy, cross-country in a RV in hopes of convincing MTV's "Pimp My Ride" to customize Darius' wheelchair.

Links:

Awards:

  • "Darius" has won 25 awards in just 25 film festivals.
  • Filmmaker Logan Smalley, a 22 year-old first-time director-producer with no previous film experience, also wrote and performed the film’s music.

Raves:

  • "Certain to stir hearts. . ." -- Variety
  • "If you get a chance to see 'Darius Goes West,' don't miss it. Your life will be touched." -- Moviefone
  • "It's a wrenching, funny and charming film guaranteed to touch any heart." -- The Oregonian



Celebrity Snow Jam (March 25-27, 2005, Seattle WA)

The 1st annual Celebrity Snow Jam is history.

We met some great people, saw some beautiful scenery, had some good times, and most importantly, raised some money for desperately needed research.

There were so many wonderful people who pitched in to make our first event such a success. We really appreciate everyone who helped out, and everybody who came to the various events.

Thanks first go to our celebrities -- and what a great group of people they were! Thanks to the following for coming and bringing their humor, forbearance, and wonderful caring attitudes along with them:

David Anders (“Alias”)
Billy Burke (“24”)
Robert Carradine (“Lizzie McGuire”)
Majandra Delfino (“Roswell”)
Don Felder (The Eagles)
Devon Gummersall (“The L Word”)
LaLaine  (“Lizzie McGuire”)
Shannon Lucio (“The O.C.”)
Jake Thomas (“Lizzie McGuire”)
Jim Turner (“Arli$$”)
And, of course, Kevin Weisman (“Alias”)

Special thanks goes to Don Felder of The Eagles, who donated a Signal snowboard that he, and the other CSJ celebrities, autographed. It’ll soon be put up for auction on eBay – watch for it!

We very much appreciate Dr. Jeffrey Chamberlain, director of the Paul Wellstone Muscular Dystrophy Research Center at the U. of Washington School of Medicine, one of the four NIH Muscular Dystrophy Centers of Excellence in the country, taking the time to come to the events, and speaking extemporaneously, and so eloquently, about DMD.

Thanks also go to John Zaring & Bianca Cioffi of Zaring/Cioffi Entertainment and their terrific staff for producing the event. We literally couldn’t have done it without them. And we’ll be doing it with them again next year – bigger and better!

Bill Boyd and Amy Striebel were invaluable. Bill did all the cinematography, producing, directing & editing for the coverage of the event, and Amy organized, ticketed, collected, and generally made herself indispensable. Thanks my friends!

Chris Pirillo of Lockergnome.com fame, conducted 2 extensive Internet interviews, one each with Kevin & me, to aid in publicizing CSJ & DMD. The interviews can be heard at www.ChrisPirillo.com. Thanks Chris (and Ponzi)!

Our wonderful volunteers, bless their hearts, were terrific –- efficient, helpful, resourceful -- thanks to all! (Please come back next year!)

And our sponsors: Shannon at Avalon Limos; Sam & his staff at Salish Lodge & Spa; Dan at Car Toys; Colleen at American Airlines; ABC-TV, Touchstone Productions & JJ Abrams & staff at “Alias;” Kathi Goertzen at KOMO-TV4; The End 107.7 radio; Larry at Tall Guy Productions; The Summit at Snoqualmie; Mark at Premier Club; Red Bull; Willie at Chef by Design; Sema at Jay Berry’s; Eric at McCormick & Schmick’s. We’d love to have you all back next year too!

And last, and most important, our heartfelt thanks to the DMD families who came and so generously and openly shared their experiences with DMD so that those of the CSJ guests, volunteers and sponsors who were unfamiliar with the toll this deadly disease takes on families could take a measure of understanding with them.  Koren Stark & Zach; Don & Yvonne Hill & Joey and their daughters -- thank you so very much for helping educate and edify us.

Congrats go to LaLaine (“Lizzie McGuire”), who won the Celebrity Poker Jam. And she’d never played before – she learned by watching the various celebrity poker games on TV! Now that takes smarts! (And you thought she was just a pretty face … and a great voice … and a talented actor … and …)

Also congrats to Jim Picicci who won the CSJ Raffle – round trip air fare for two to LA, hotel stay, lunch with Kevin Weisman on the “Alias” lot, LOTS of cool “Alias” stuff (all signed by the whole “Alias” cast), tickets to the “Jimmy Kimmel Live” VIP Green Room and show taping, assorted “Jimmy Kimmel” stuff – not a bad deal for a $10 raffle ticket!  

Thanks again Seattle!



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Kyle Kyllan • • PO Box 17371 - Encino, CA 91416 • 818-692-5500