A head-to-head trial comparing the decades-old steroid, prednisone, and a promising new steroid, vamorolone, finds both act on the same key set of genetic pathways involved in controlling inflammation, indicates a new study ...
Sep 20, 2018
0
4
Duchenne is the most common and severe form of muscular dystrophy. Because of this genetic disease, one out of every 3,500 children spends their 12th birthday in a wheelchair. This disorder progressively leads to general ...
Oct 19, 2016
0
205
Using the new gene-editing enzyme CRISPR-Cpf1, researchers at UT Southwestern Medical Center have successfully corrected Duchenne muscular dystrophy in human cells and mice in the lab.
Apr 12, 2017
0
226
Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR) and the Royal Holloway University of London demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, ...
Jul 25, 2017
0
343
Regenerative medicine researchers at UT Southwestern Medical Center developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy.
Nov 30, 2017
0
195
A promising therapy for Duchenne muscular dystrophy (DMD) developed by University at Buffalo researchers is moving closer to use in humans.
Oct 2, 2018
0
60
A team of researchers from the U.S. and Germany describes a novel CRISPR approach to produce healthy heart muscle using pluripotent stem cells from Duchenne muscular dystrophy (DMD) patients. In their paper published on the ...
Stanford University School of Medicine researchers have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which ...
Sep 3, 2018
0
118
Researchers at The Ohio State University Ross Heart Hospital and Nationwide Children's Hospital have shown early treatment with the heart failure medication eplerenone can improve heart function in young boys with Duchenne ...
Mar 7, 2017
0
326
A new study published in the journal Nature Communications describes a breakthrough in research related to facioscapulohumeral muscular dystrophy (FSHD). The debilitating genetic disease - which has no approved treatment ...
Sep 15, 2017
0
187
