- CONNECT1-EDO51 will assess dystrophin levels, exon skipping data and safety data following multiple doses of PGN-EDO51 in patients with mutations amenable to skipping exon 51 - - Initial data. | May 18, 2023
with brecken stopped going upstairs and they then had some hope of an experimental gene therapy of a one-time trial. the idea is that it is caused by a gene, and if you can take a normal working gene and put it back into the muscles of the kids, it should fix the gene. reporter: it is like a dystrophin. and then it should repair itself. reporter: in trials it did that, and the families like his are convinced he is walking better and staying stronger for years longer than he would have. but a key maker of the drug s maker did not meet one of the drug s goals, and that did not make to a certain path to
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne muscular dystrophy (DMD).
In a new study, the group of Johan Auwerx at EPFL's School of Life Sciences has made the first connection between muscular dystrophy and sphingolipids, a group of bioactive lipids. The study is published in Science Advances.