| Kabbara's breakthrough is a treatment for Metachromatic Leukodystrophy which is a genetic disorder that affects nerves, muscles and other organs. |
| Myelin-associated glycoprotein is altered in a familial late-onset orthochromatic leukodystrophy. |
| In the second trial, Naldini and his colleagues treated three children with a metabolic disease called metachromatic leukodystrophy. |
| Case 2 involved a 3-year-old boy who received a mismatched unrelated bone marrow transplant for metachromatic leukodystrophy. |
| The classic leukodystrophies include adrenoleukodystrophy, Krabbe's globoid cell, and metachromatic leukodystrophy, and a few other less well known entities. |
