Leukodystrophy refers to a group of diseases that affect the central nervous system. Learn the causes, symptoms, and treatment options of Leukodystrophy today. Skip to main content .

Leukodystrophy damages the brain’s white matter, which is made up of nerve fibers and a protective layer called the myelin sheath. Different types of genetic changes cause each type of ...

Genetic white matter disorders (inherited leukodystrophies) affect the myelin—or white matter—of the brain, spinal cord, and often peripheral nerves. Myelin is the protective covering of the nerve ...

A 3 year-old girl presented with progressive spasticity, visual loss and loss of motor milestones. Metachromatic Leukodystrophy (MLD): Flair axial MRI images. Note the bilateral confluent white matter ...

Orchard Therapeutics hopes its newly approved treatment for metachromatic leukodystrophy will save children's lives — and establish that this form of gene therapy can be profitable.

Walking speed is gaining traction as a vital sign, providing critical information about physical and mental health risks. Here’s when to get a gait analysis.

Neurology > General Neurology Pediatric Neuron Disease Gets Its First Gene Therapy — FDA approves arsa-cel for metachromatic leukodystrophy. by Judy George, Deputy Managing Editor, MedPage Today ...

Metachromatic leukodystrophy, MLD for short, is a rare but highly debilitating neurodegenerative disease. It is noteworthy for its difficulties and the potential promise the latest research offers ...

A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a wholesale price of $4.25 million, its manufacturer announced Wednesday, making it the world’s ...

Officials say metachromatic leukodystrophy (MLD) will likely be added to the list of conditions Minnesota newborns are routinely screened for in 2026, based on the Newborn Screening Advisory ...