Duchenne Muscular Dystrophy
For generations, a Duchenne diagnosis was a final verdict. There were no meaningful treatments. But now multiple companies are developing gene therapies - Andrew Joseph
image by: CureDuchenne
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Lacking Cure, a New Tack on a Muscle Disease
But for all the money collected toward a cure, Duchenne muscular dystrophy, the most common form of the disease that afflicts children, still confines thousands of boys in this country to wheelchairs in their early teens. Many do not live past their 20s...
The approach is changing at a few medical centers, which are focused on making better use of available therapies to eke out longer lives for their patients. Rather than concentrate only on a cure, some researchers are now intent on developing drugs that may alleviate the effects of the disease. But, absent a cure, too many doctors around the country still assume there is little or nothing that can be done for the muscle-wasting…
Resources
FDA advisers narrowly back first gene therapy for muscular dystrophy
Patients with Duchenne muscular dystrophy are missing the muscle-protecting protein or don't make enough of it. While not a cure, Sarepta argues that its "micro-dystrophin" treatment can help slow the progression of the disease.
Being Rare
His rareness means he cannot run, jump, hop, walk at normal speed, climb in the playground, swing on a swing……I won’t go on.
Duchenne Has Forced Me to Reflect on My Own Mortality
It was quite easy for me to spot differences between my friends who grew up without DMD and me. I looked weird when I walked or ran. Once puberty hit, they got muscular and started growing facial hair, and I did not.
CRISPR Offers New Line of Attack on Muscular Dystrophy
The technique could help up to 60 percent of those suffering from a specific type of dystrophy known as DMD.
A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived
This is the story of a fatal genetic disease, a tenacious scientist and a family that never lost hope. Conner Curran was 4 years old when he was diagnosed with Duchenne muscular dystrophy, a genetic disease that causes muscles to waste away.
A Gene Therapy Appears To Replace Missing Protein In Muscular Dystrophy Patients
An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy, a deadly and irreversible disease...
Defying the Odds of Living with Duchenne, Decade After Decade
When it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the lucky ones. A resident of São Paulo, Brazil’s largest city, Feder was diagnosed with Duchenne at age 6 and immediately began taking corticosteroids as well as heart medicine and supplements.
FDA changes course on Sarepta’s once-rejected muscular dystrophy drug
The Food and Drug Administration reversed its decision on a treatment for Duchenne muscular dystrophy from Sarepta Therapeutics, approving a previously rejected drug without explaining what the problem was in the first place.
For Many Boys With Duchenne Muscular Dystrophy, Bright Hope Lies Just Beyond Reach
Scientists are testing nearly two dozen treatments that might stop the disease. But enrollment in the trials is very restricted, and few children qualify.
Gene Editing Offers Hope for Treating Duchenne Muscular Dystrophy, Studies Find
After decades of disappointingly slow progress, researchers have taken a substantial step toward a possible treatment for Duchenne muscular dystrophy with the help of a powerful new gene-editing technique.
How Gene Therapy Helped Conner Run
Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, it could change the lives of thousands of children with Duchenne muscular dystrophy.
They rallied around ‘our boys’ as they pushed for a Duchenne cure. Where did that leave girls?
For generations, a Duchenne diagnosis was a final verdict. There were no meaningful treatments. But now multiple companies are developing gene therapies that, if they are shown to be effective, could help patients make a form of the protein they’re lacking, fundamentally altering their lives. For maybe the first time ever, there is genuine excitement in the Duchenne field. Families of girls affected by the disease, however, fear the advances might pass them by.
Lacking Cure, a New Tack on a Muscle Disease
But for all the money collected toward a cure, Duchenne muscular dystrophy, the most common form of the disease that afflicts children, still confines thousands of boys in this country to wheelchairs in their early teens. Many do not live past their 20s. It is a stark reminder of how American medicine with its focus on breakthrough treatments can sometimes fail a complex, rare and stubbornly uncurable disease. Single-minded in their pursuit of a cure, doctors and researchers for years all but ignored the necessary and unglamorous work of managing Duchenne (pronounced doo-SHEN) as a chronic condition.
My Boy, Duchenne & Me
Not just life as a mother, but an unexpected one as a mother of a son with Duchenne Muscular Dystrophy.
Cure Duchenne
CureDuchenne is a national nonprofit that raises awareness and funds to find a cure for Duchenne muscular dystrophy. For the first time ever there is hope to find a cure for this destructive disease affecting thousands of boys and their families.
Duchenne Muscular Dystrophy Research Fund
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.
Fighting DMD
Duchenne is a genetic disease and boys are historically diagnosed by the ages of 5 to 6 years old. My wife and I started the Fighting Duchenne Foundation to fight for kids all over the world that can’t fight for themselves. Without your help, over 200,000 kids born with Duchenne, including our 2 sons will not have the possibility of enjoying a long and healthy life.
Foundation to Eradicate Duchenne
Welcome to the Foundation to Eradicate Duchenne, a 501c(3) organization established in 2002 with the goal of finding treatments and an ultimate cure for Duchenne Muscular Dystrophy, the world’s leading lethal childhood genetic disease.
Jett Foundation
Since 2001, the Jett Foundation, located in Kingston, MA, has worked to find treatments and a cure for Duchenne muscular dystrophy while improving the lives of those affected by DMD. Over the past decade, the Jett Foundation has raised almost 2 million dollars for Duchenne research. The foundation currently funds the Jett Program for Pediatric Neuromuscular Disorders at Mass General Hospital for Children. The Jett Foundation provides resources for treatments and adaptive activities as we work toward a cure and treatments for Duchenne.
Parent Project Muscular Dystrophy
PPMD is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy–our mission is to end Duchenne. We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.
Suneel's Light
The foundation was established to fund critical research for treatments and a cure for Duchenne Muscular Dystrophy, the most lethal genetic childhood disease which targets young boys, without regard to ethnicity, and leads to death from respiratory or heart failure by their early 20's. Without a breakthrough in ongoing medical research and treatment, there is little hope that these children, will live to adulthood.
National Human Genome Research Institute
Duchenne muscular dystrophy affects approximately 1 in 3500 male births worldwide. Because this is an inherited disorder, risks include a family history of Duchenne muscular dystrophy.
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