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 ...

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, ...

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 ...

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 ...

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 ...

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 ...

The approval makes atidarsagene autotemcel (arsa-cel [Lenmeldy]; Orchard Therapeutics) the first approved treatment for metachromatic leukodystrophy, a disease caused by a mutation in the ARSA gene ...

If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic ...

In what is being described as a "breakthrough moment", the NHS has provided for the first time a life-saving gene therapy treatment for the fatal disorder metachromatic leukodystrophy to a baby.