welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- log in
For Many Boys With Duchenne Muscular Dystrophy, Bright Hope Lies Just Beyond Reach
Lucas was 5 before his parents, Bill and Marci Barton of Grand Haven, Mich., finally got an explanation for his difficulties standing up or climbing stairs. The diagnosis: muscular dystrophy.
Mr. Barton turned to Google.
“The first thing I read was, ‘no cure, in a wheelchair in their teens, pass in their 20s,” Mr. Barton said. “I stopped. I couldn’t read any more. I couldn’t handle it.”
Then he found a reason to hope. For the first time ever, there are clinical trials — nearly two dozen — testing treatments that might actually stop the disease.
The problem, as Mr. Barton soon discovered, is that the enrollment criteria are so restrictive that very few children qualify. As a result, families like the Bartons often are turned away.
rareRelated
-
Navigating School for Individuals with DuchenneSchool can be an exhausting experience f...
-
Discovery Could Lead to Improved Therapies for Duchenne Muscular Dystrophyhttps://www.youtube.com/watch?v=TzoqfAak...
-
Powered standing wheelchairs promote independence, health and community involvement in adolescents with Duchenne mus...Save Our Sons Duchenne Foundation is pro...
-
The Burden of Access in Duchenne Muscular Dystrophy in the USThis study is being conducted to determi...
-
Antisense Oligonucleotides for the Treatment of Cardiomyopathy in Duchenne Muscular DystrophyDuchenne muscular dystrophy (DMD) is an ...
-
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystroph...The proposed clinical trial is an outgro...
-
ICER Releases Draft Evidence Report on Treatments for Duchenne Muscular DystrophyThe Institute for Clinical and Economic ...
