The world can be overwhelming thanks to the deadly coronavirus pandemic, especially for someone dealing with muscular dystrophy. On top of the daily struggles — lack of mobility, physical exhaustion, and steroid-related side effects — there’s also the worry of getting sick and feeling the virus’s financial toll.
The technique could help up to 60 percent of those suffering from a specific type of dystrophy known as DMD.
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.
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.
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 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.
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...
Scientists are testing nearly two dozen treatments that might stop the disease. But enrollment in the trials is very restricted, and few children qualify.
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.
While there is no universal pattern that will work in every scenario for a student with either type of Muscular Dystrophy, the general requirements for choosing a school include...
There's hope for patients with myotonic dystrophy. A new small molecule developed by researchers at the University of Illinois has been shown to break up the protein-RNA clusters that cause the disease in living human cells, an important first step toward developing a pharmaceutical treatment for the as-yet untreatable disease.
Muscular dystrophy is a group of inherited diseases that are characterized by weakness and wasting away of muscle tissue, with or without the breakdown of nerve tissue. There are nine types of muscular dystrophy, with each type involving an eventual loss of strength, increasing disability, and possible deformity.
My parents tried to shield me as long as they could from data that revealed the average life expectancy of patients with Duchenne muscular dystrophy (DMD).
Muscular Dystrophy News is a digital news publication dedicated to offering comprehensive daily news coverage of MD.
Cure CMD’s mission is to bring research, treatments and in the future, a cure for the Congenital Muscular Dystrophies that have a profound affect on children as young as birth.
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.
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.
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.
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.
We are the world's largest and most progressive grassroots network of facioscapulohumeral muscular dystrophy patients, their families and research activists. The FSH Society harnesses the power and insight of a patient-driven model, as it was founded on a promise between two research scientists with FSHD never to let the disease be forgotten or neglected. We are a cause without borders.
The Jain Foundation's mission is to cure muscular dystrophies caused by dysferlin protein deficiency. These dystrophies are collectively termed dysferlinopathy, and include Limb-girdle muscular dystrophy type 2B and Miyoshi muscular dystrophy 1. Dysferlinopathy is an orphan disease afflicting approximately 5 people/million and receives little or no funding from traditional sources.
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.
The Muscular Dystrophy Association is a voluntary health agency -- a dedicated partnership between scientists and concerned citizens aimed at conquering neuromuscular diseases that affect more than a million Americans.
Muscular Dystrophy Canada is a not-for-profit organization committed to finding a cure for neuromuscular disorders through well funded research. Muscular Dystrophy Canada’s dedicated volunteers and staff across the country raise funds to enhance the lives of those affected by over 150 different kinds of neuromuscular disorders by continually working to provide ongoing support and resources.
Through the life services of Advocacy, Education and Consultation, Muscular Dystrophy Family Fund will enhance and strengthen the lives of individuals with neuromuscular diseases and to provide, or direct their families to valuable resources in helping our clients become successful contributors to the community.
We aim to find treatments and ultimately cures for muscular dystrophy and related conditions and improve the lives of all those people affected by them in the UK. We act to make a real difference for people affected by muscular dystrophy and related conditions.
The Myotonic Dystrophy Foundation is a non-profit organization founded in 2007 by families living with myotonic dystrophy. Through community support, education, advocacy, and research MDF is committed to enhancing the quality of life of people living with myotonic dystrophy, and maximizing efforts focused on treatments and a cure for DM.
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.
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.
Coalition to Cure Calpain 3 provides support for promising research into finding treatments or a cure for limb-girdle muscular dystrophy, type 2A/Calpainopathy (LGMD2A). The unrelenting nature of this disease takes its victims from full mobility to a wheelchair within 11-28 years after the onset of symptoms. LGMD2A attracts significantly fewer research dollars than other forms of muscular dystrophy and thus fewer researchers working to understand the disease and discover a cure.
Join Brad Miller who was diagnosed with Becker Muscular Dystrophy at just 10 years old, a condition which limits his ability to walk and gets progressively worse over time. He created the My Becker's Story blog in 2010 to share my story and to help raise awareness about Muscular Dystrophy.
Essays by Mark E. Smith, WheelchairJunkie.com
The Paul D. Wellstone Muscular Dystrophy Cooperative Research Center is exploring therapeutic strategies for the treatment of various muscular dystrophies by enabling translational research on muscular dystrophies and providing advanced diagnostic services. The MDCRC is composed of two research projects, three cores and investigators with a proven track record of excellence and collaboration.
Muscular dystrophies are a group of disorders that result in muscle weakness and a decrease in muscle mass over time. Some muscular dystrophies are not identified until a child is 3 to 6 years of age, but can be diagnosed earlier.
Muscular dystrophy is a genetic disorder that gradually weakens the body's muscles. It's caused by incorrect or missing genetic information that prevents the body from making the proteins needed to build and maintain healthy muscles.
In depth information about muscular dystrophy including further resources.
Information, research, clinical trials, articles and resources for muscular dystrophy from MedlinePlus.
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.
Muscular dystrophy (MD) is a genetic (inherited) condition that gradually causes the muscles to weaken. This leads to an increasing level of disability.
The muscular dystrophies are a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in infancy or childhood, while others may not appear until middle age or later.