What is ALD?
Adrenoleukodystophy (ALD) is a rare, inherited genetic disorder that primarily affects boys and young men. In this disorder, the myelin sheath covering the nerves in the brain is destroyed and the adrenal gland degenerates. As a result, individuals with ALD gradually lose brain functioning eventually resulting in a total vegetative state and eventually death. The average time from diagnosis to a complete vegetative state is approximately 2 years.
For children diagnosed with ALD, time is of the essence. Asymptomatic children may be eligible to participate in clinical trials of "Lorenzo's Oil" which has been shown to slow the progression of symptoms. For children who have already developed symptoms, bone marrow or umbilical cord blood transplantation seems to be the best option. However, because ALD is so rare, results vary for each patient.
To learn more about adrenoleukodystrophy and related disorders, please visit the links below.
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Suggestions for Parents of Children with ALD
Zachary's dad, Carl, would like to encourage parents of children who have been diagnosed with ALD to pursue transplant options immediately. Unfortunately, in Zachary's case, waiting for the results of a skin biopsy turned into wasting precious time. There are currently three locations in the United States that conduct bone marrow or umbilical cord blood stem cell transplants for children with ALD. Contact information for the transplant professionals is as follows:
Children's Mercy Hospital
Kansas City, MO
Dr. Charles Peters, Director of Hematopoietic Stem Cell Transplantation
816-234-3265
Duke University Pediatric Blood and Bone Marrow Transplant Program
North Carolina
Dr. Joanne Kurtzberg, Founder and Director
919-668-9100
University of Minnesota Children's Hospital
Minneapolis, MN
Dr. Lawrence Charnas, M.D., Ph.D
612-625-7466
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