A failed phase 3 trial for a Pfizer treatment announced several months later cast more gloom over the gene therapy approach in DMD. At the heart of DMD is the breakdown of the protein dystrophin ...

Gene sequencing specialist PacBio has reached an agreement to buy Apton Biosystems, boosting its position in short-read sequencing technology that has traditionally been dominated by rival Illumina.

“Finally, we may be there. I am very hopeful. This is an emotional time for people in the field.” Duchenne muscular dystrophy (DMD) is a rare genetic disorder caused by loss-of-function mutations in ...

Without functional dystrophin to support muscle strength and stability, muscle fibers are easily damaged. Duchenne muscular dystrophy results from mutations in the DMD gene that encodes the dystrophin ...

Topic sponsors are not involved in the creation of editorial content. "Pfizer’s Phase III DMD gene therapy trial misses primary endpoint" was originally created and published by Clinical Trials ...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...