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Research is currently being done by several groups on MLD:

  • Shire has announced that it will begin enrolling patients in a natural history study for enzyme placement therapy in 2007.  Click here for more details.

  • Dr. Joanne Kurtzberg and Dr. Donald Beam are working on a cell replacement therapy at Duke University.  As of mid-2007, this research was being reviewed by the FDA.
  • Zymenex, a Danish company, is currently working on an enzyme replacement drug/therapy called Metazym.  As of October 2005, it estimated that clinical trials may begin at the end of 2006.  Dr. Belle Humphrey of the UK division of The Myelin Project has also met with Zymenex in conjunction with this research.
  • A team at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy is working on gene therapy research to deliver the ARSA enzyme to tissues affected by MLD.  A new article on cell replacement therapy from this same group appeared in The Journal of Neuroscience on March 22, 2006.


  • An article from e-Medicine that describes MLD in detail (may require you to log in, but it's free).


  • In December 2007, Dr. Vinod Prasad of Duke University presented a paper describing how cord blood transplants are an effective treatment for children with metabolic disorders, such as MLD.  However, the children must be treated before significant symptoms appear.  Click here for the full article.


  • On December 20, 2005, President George W. Bush signed into law a bill establishing a national umbilical cord blood program providing federal funding to collect and store cord blood for life-saving blood cell transplants and reauthorizing the existing national registry for marrow donors.  The combined program has been named the C.W. Bill Young Cell Transplantation Program.  The bill will provide $79 million in federal funding to increase the number of cord blood units available for matches. The goal is to provide an additional 150,000 cord blood units for public use and establish a system that allows transplant physicians access to adult volunteer donors and cord blood units.  For the full article, click here.   


  • Link to a video of Dr. Joanne Kurtzberg discussing stem cell transplants (click on Dr. Joanne Kurtzberg on the page that appears).


  • Article describing Dr. Joanne Kurtzberg's cord blood transplant program at Duke.
  • What is Cord Blood?
  • This article on umbilical cord blood discusses the benefits of cord blood donation, discusses the donation process and reviews private vs. public donation


  • The Trimper Family has three children affected by MLD and has been instrumental in gaining support and generating funds to support MLD research.  View their website at www.trimperchildren.org.
  • Founded by Augusto Ordone of Lorenzo's Oil fame, The Myelin Project focuses on myelin-related disorders.
  • Emma's Foundation donates to research programs helping with treatments or potential cures for MLD.
  • Hunter's Hope was founded by former Buffalo Bill quarterback Jim Kelly after his son,  Hunter, was diagnosed with Krabbe's Disease, which is another leukodystrophy. Click here to read an article on Hunter and their organization's efforts.
  • The MLD Foundation offers an e-mail support group for families affected by MLD.