Leukodystrophy refers to a group of rare genetic disorders affecting the central nervous system (CNS). The CNS includes the brain and spinal cord. The outlook for people with these disorders is ...

Leukodystrophy isn’t just one disease; it’s actually a group of diseases that affect the central nervous system. Doctors are discovering new forms of leukodystrophy all the time, but experts currently ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

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 ...

Please provide your email address to receive an email when new articles are posted on . Biotech firm SynaptixBio has entered into a licensing agreement with the Children’s Hospital of Philadelphia, ...

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The FDA approved ...

Even now, only a portion of leukodystrophy patients are correctly diagnosed, though various causative genes have been identified. In the present report, we describe a case of adult-onset ...

THE WOODLANDS, Texas (KTRK) -- A family in The Woodlands is seeking help after two of their children were diagnosed with a deadly medical disorder. "It's so hard on their little bodies," said mother ...

Finding showed treatment with Lenmeldy significantly extended severe motor impairment-free survival in children with PSLI MLD compared with untreated natural history children. The Food and Drug ...

TUBB4a leukodystrophy is an "incurable genetic condition" that affects one in 1,663 births, according to the Cleveland Clinic. First identified in 2014, it makes up a small percentage of the 50 rare ...

Live your life healthier and happier with our free weekly Living Well newsletter A scientist who diagnosed her own daughter with an incurable genetic disorder has set up a foundation to raise funds ...