Judith van Deutekom

A multinational pharmaceutical company has committed to developing Prosensa's exon skipping compound for DMD

posted on October 19, 2009 - 9:00pm
On Oct. 13, 2009, the multinational pharmaceutical company GlaxoSmithKline announced it will develop and commercialize the experimental “exon skipping” compound PRO051 for Duchenne muscular dystrophy (DMD). The commitment of a major pharmaceutical company to development of a drug for DMD is very good news for families with this disease.
posted on March 1, 2008 - 10:21am
QUEST Vol. 15, No. 2
A European group of researchers successfully used a combination of genetic correction and stem cells to treat Duchenne muscular dystrophy (DMD) in mice with the disease. The compound they used for the genetic correction was developed by Judith van Deutekom, then at Leiden (Netherlands) University Medical Center, with MDA support. DMD results from the loss of the muscle protein dystrophin because...
posted on July 1, 2007 - 10:32am
QUEST Vol. 14, No. 4
The underlying cause of Duchenne muscular dystrophy (DMD) can be any of a large number of mutations in the gene for the dystrophin protein. One strategy now being tested for overcoming this problem is gene therapy, the insertion of new dystrophin genes. Another is the transplantation of cells that give rise to muscle.