Adrenoleukodystrophy (ALD) Research Fund
Please join us in supporting the Leukodystrophy Program at the University of Minnesota. Dr. Paul Orchard, Dr. Troy Lund, and Dr. Weston Miller are experts in the field of Pediatric Blood and Marrow Transplantation and have unsurpassed experience treating patients with adrenoleukodystrophy (ALD) from all around the world. We are inspired by the work they are doing, so we launched this campaign in an effort to accelerate their research and empower them to translate their ideas into a cure.
ALD is an x-linked disorder caused by mutations in the ABCD1 gene. Boys with adrenoleukodystrophy are unable to metabolize very long chain fatty acids (VLCFA). The resulting build-up of VLCFA can cause loss of function in the adrenal glands and testes. In some boys, the central nervous system is affected whereby the myelin sheaths that protect nerves are destroyed. Normally, nerve endings are covered with a myelin sheath which helps speed the communication of nerve impulses. If myelin is lost, the underlying nerve cell will become dysfunctional and die. When this breakdown occurs in the brain, it's known as cerebral ALD, which often causes rapidly progressive neurologic deterioration and eventual death. The only effective treatment option for boys and adolescents with early-stage cerebral ALD is hematopoietic cell transplantation (HCT).
Early diagnosis of ALD is the key to saving lives. Newborn screening allows prospective monitoring for adrenal function and the onset of cerebral ALD. It presents a new opportunity to research many aspects of the disease. In February 2016, ALD was added to the United States Recommended Uniform Screening Panel. Since then, Minnesota and other states have implemented newborn screening for ALD or have initiated processes intended to add ALD to their newborn screening program.
The money raised in this campaign will be used to help the team discover methods for earlier detection of the cerebral form of adrenoleukodystrophy. They are investigating important clinical questions related to the initiation of disease, oxidative stress, and genotype-phenotype correlations. It will also allow them to launch additional research projects that will help them better understand the basic biology of adrenoleukodystrophy and the role of HCT as an intervention. Our ultimate goal is to help them develop safer, more effective therapies that will improve outcomes and save lives.
Please contact Kelly Miettunen with questions or for additional information. She can be reached at firstname.lastname@example.org.
|Adrenoleukodystrophy Research Fund||Raised|
|Olga Diner||$ 250.00|