This observational study aims to better understand rare genetic disorders that affect the brain, such as MLD, Krabbe Disease, ALD, MPS I, and MPS II. Researchers are collecting information about how these diseases naturally progress (Palliative Care) and are also following patients who have received Hematopoietic Stem Cell Transplantation (HSCT) as part of their regular medical care. The study is looking to see how these conditions affect cognitive (thinking and learning), language, and gross motor (large muscle movement) development over 15 years. Anyone with a genetic neurodegenerative disorder can participate. The current recruitment status is unclear.

Study design

This is an observational study with a planned enrollment of 1500 participants. It is not a treatment study, but rather collects information over time.

What's involved

You would be evaluated by a team of specialists every 3 months for the first year, every 6 months for the second year, and once a year after that.

Compensation

Not stated in the trial record.

Follow-up

Participants will be followed for 15 years to assess cognitive, language, and gross motor development.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.