- Familial Xanthomatosis
- Liposomal Acid Lipase Deficiency, Wolman Type
Your QuestionAre there any new therapies for Wolman disease? Is it curable? How can I find clinical trials and research studies?
We have identified the following information that we hope you find helpful. If you still have questions, please contact us.
Questions on this page
- What is Wolman disease?
- Is Wolman disease curable?
- Are there any clinical trials enrolling patients with Wolman disease?
- How can I find transplant centers that are treating children with Wolman disease?
- Are other therapies for Wolman disease being investigated?
- How can I find additional comprehensive information on the treatment of Wolman disease?
Click here to learn more about hematopoietic cells.
Click here to learn more about bone marrow transplantation.
Click here to learn more about umbilical cord stem cell transplantation.
You can also contact the Patient Recruitment and Public Liaison (PRPL) Office at the National Institutes of Health (NIH). We recommend calling the toll-free number listed below to speak with a specialist, who can help you determine if your child is eligible for any clinical trials.
Patient Recruitment and Public Liaison Office
NIH Clinical Center
Bethesda, Maryland 20892-2655
Web site: http://clinicalcenter.nih.gov/
If you are interested in enrolling your child in a clinical trial, you can find helpful general information on clinical trials at the following ClinicalTrials.gov Web page.
A tutorial about clinical trials that can also help answer your questions can be found at the following link from the National Library of Medicine:
Resources on many charitable or special-fare flights to research and treatment sites and low-cost hospitality accommodations for outpatients and family members, as well as ambulance services, are listed on the Web site of the Office of Rare Diseases (ORD), part of the National Institutes of Health.
The National Library of Medicine (NLM) Web site has a page for locating libraries in your area that can provide direct access to these journals (print or online). The Web page also describes how you can get these articles through interlibrary loan and Loansome Doc (an NLM document-ordering service). You can access this page at the following link http://nnlm.gov/members/. You can also contact the NLM toll-free at 888-346-3656 to locate libraries in your area.
- Wolman disease. Genetics Home Reference. October 2007; http://ghr.nlm.nih.gov/condition/wolman-disease.
- Kruer MC, Steiner RD. Lysosomal Storage Disease: Wolman Disease and Cholesteryl Ester Storage Disease. Medscape Reference. October 18, 2013; http://emedicine.medscape.com/article/1182830-overview#a7.
- Tolar J, Petryk A, Khan K, Bjoraker KJ, Jessurun J, Dolan M, Kivisto T, Charnas L, Shapiro EG, Orchard PJ. Long-term metabolic, endocrine, and neuropsychological outcome of hematopoietic cell transplantation for Wolman disease. Bone Marrow Transplant. 2008 Sep 8;
- Stein J, Garty BZ, Dror Y, Fenig E, Zeigler M, Yaniv I. Successful treatment of Wolman disease by unrelated umbilical cord blood transplantation. Eur J Pediatr. 2007 Jul;
- Du H, Cameron TL, Garger SJ, Pogue GP, Hamm LA, White E, Hanley KM, Grabowski GA. Wolman disease/cholesteryl ester storage disease: efficacy of plant-produced human lysosomal acid lipase in mice. J Lipid Res. 2008 Aug;