ISMRD
The 2012 International Conference for Glycoprotein Storage Diseases and the extension of the Natural History Study will take place in Charleston South Carolina July 27th - July 30th 2012.
Therapies
Current therapies for Glycoprotein Storage Diseases are primarily supportive or palliative in nature. However, bone marrow and cord blood transplantation have shown initial success for patients with Alpha-Mannosidosis, Fucosidosis and, potentially, Mucolipidosis II. Enzyme Replacement Therapy is currently available for a few LSD's and is being explored for Alpha-Mannosidosis.
Bone Marrow & Cord Blood Transplants
- BMT FAQ: The University of Minnesota's resource for bone marrow transplants
- BMT Resources: Information assembled for the lay person by a five-time transplant recipient
- Center for International Blood and Marrow Transplant Research
- Cord Blood Donor Foundation
- Genetic Disease Treatment Options: University of California San Francisco transplant program's information on options for Lysosomal Diseases
- National Cord Blood Program
- National Marrow Donor Program
- Robert's Road to Recovery: Kathleen King's online journal about her son, Robert's, two transplants for Alpha-Mannosidosis.
- Ronald McDonald House Charities: Ronald McDonald Houses provide housing support internationally for families with a relative receiving a transplant
- Transplant Center Search: find a North American transplant center via BMT Infonet
- TransWeb
Enzyme Replacement Therapy (ERT)
- Efficacy of ERT In Mice with Alpha-Mannosidosis
- The Hue-Man Project: A European collaborative effort to develop an effective enzyme replacement therapy for Alpha-Mannosidosis
- Zymenex: Scandinavian biopharmaceutical company currently engaged in the late stage pre-clinical development of a pharmaceutical intervention for Alpha-Mannosidosis