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
-
Dear DuchenneDear Duchenne, It’s been less than tw...
-
A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy (REACH-DMD)The purpose of this study is to evaluate...
-
How Facebook and Twitter could be the next disruptive force in clinical trialsAmber Sapp was browsing the Internet lat...
-
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingThis is a multicenter, open-label, dose-...
-
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyThis is an open-label study to assess th...
-
Their Voice: Student, mentor share triumph at graduationAt the age of 8, Porter Aydelotte and hi...
-
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrop...it is a randomised, double blind, parall...
-
Understanding Neuromuscular Disease CareNeuromuscular diseases (NMD) encompass a...
-
A Mother’s Guilt – Finding a Way Through the DarknessI Am a Carrier I’m a carrier of many ...
