welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
How Facebook and Twitter could be the next disruptive force in clinical trials
Amber Sapp was browsing the Internet late one night in August when she happened to find out that her 12-year-old son’s clinical trial had failed.
Every four weeks for two-and-a-half years, she had shuttled Garrett to a hospital nearly six hours away. There, he was prodded and pricked with needles in the hope that the antibody treatment being tested would reverse a devastating genetic disease called Duchenne muscular dystrophy. But an early data analysis, Sapp learnt, had shown that the treatment wasn’t working.
The thought of wasting Garrett’s limited time with a failed trial was hard enough. The news was all the more disturbing because it didn’t come from the trial organizers, but through a Facebook post from another parent. “It was upsetting that we found out that way,” says Amber. “It sent everybody on Facebook into a tizzy.” Even Garrett’s local clinical-trial coordinator, someone who should have had intimate knowledge of what was happening with the research, hadn’t yet heard the news.
rareRelated
-
Edward Clinton Smith, MDEdward C. Smith co-directs the Duke Chil...
-
John Brandsema, MDJohn Brandsema, MD, is a pediatric neuro...
-
Design of a phase 3 trial to evaluate the long-term efficacy and safety of ataluren in patients with nonsense mutati...Ataluren is conditionally approved by th...
-
Nationwide Children’s HospitalNationwide Children's Hospital is creati...
-
Hanns Lochmuller, PhDDr. Hanns currently works as a Professor...
-
Coalition DuchenneThe mission of Coalition Duchenne is to ...
-
2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy ChildrenCompare systolic function of left ventri...
