What research is taking place on ALD and AMN?
Stem cell transplants bone marrow and cord blood Scientists are studying ways to make stem cell transplants safer and beneficial for more patients. One group at the University of Minnesota has used a combination of stem cell transplantation and therapy with a drug called Mucomyst, approved by the FDA to treat cystic fibrosis and acetaminophen overdose. This was tried on three boys whose disease had already progressed past the point when bone marrow transplant is generally thought to be effective. So far, MRI scans show no further progression of the disease, and the approach is being tried in additional patients. (Bone Marrow Transplantation (2007) 39, 211215. doi:10.1038/sj.bmt.1705571) Gene therapy? Gene therapy is a new approach to stem cell transplantation. Instead of finding a donor who is enough of a match to your child to donate bone marrow stem cells or umbilical cord blood stem cells, the transplant is done using the childs own cells. The appropriate cells are removed from the
