welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
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
-
Gene editing emerges as a new therapeutic strategy for Duchenne muscular dystrophyAdvances in gene editing technology have...
-
Ataluren: An Overview of Clinical Trial Results in Nonsense Mutation Duchenne Muscular DystrophyObjective: Provide an overview of atalu...
-
Gene Therapy for Duchenne Muscular DystrophyGene Therapy for Duchenne Muscular Dyst...
-
RotM: Interview with Prof. Steve WinderFor our recent Researcher of the Month, ...
-
Israel’s ‘Little Steps’ Helps Duchenne Boys Catch the WavesOn a recent summer morning off the coast...
-
Whole Body Vibration Therapy in Boys With Duchenne Muscular DystrophyWhole-body vibration therapy (WBVT) is a...
-
Safety and Tolerability of Ataluren in a Phase 3 Study of Patients with Nonsense Mutation Duchenne Muscular Dystroph...Objective: Examine the safety/tolerabil...