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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Bone Marrow Transplant

What is Bone Marrow Transplant?

A bone marrow transplant (BMT), replaces an affected person's bone marrow with donor marrow that can produce the missing enzyme. The procedure is risky, invasive and has had very little success in Tay-Sachs, Sandhoff and GM-1. If done early enough it can be effective in slowing and even stopping the disease in other allied diseases like Krabbe and MPS.

What is the Current Status of Bone Marrow Transplant?

Currently only two centers in the US will consider Tay-Sachs, Sandhoff and GM-1 patients for BMT: Duke University Medical Center and University of Minnesota. The Duke program is not an approved clinical trial (contact June Allison-Thacker at This email address is being protected from spambots. You need JavaScript enabled to view it. to learn more).

What are the Challenges of Bone Marrow Transplant?

Bone Marrow Transplant is very invasive. Children often succumb to complications of the procedure. The immune system must be completely destroyed before introducing the healthy bone marrow. It is unclear how many of the healthy stem cells cross into the central nervous system.

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