Blood and Marrow Transplant Clinic

Adrenoleukodystrophy (ALD)

Pediatric bone marrow transplant physicians from University of Minnesota Physicians provide groundbreaking treatment for Adrenoleukodystrophy (ALD) using hematopoietic cell transplantation (HCT), commonly known as blood or marrow transplant (BMT). These specialists are part of the BMT Program at University of Minnesota Amplatz Children’s Hospital, the most experienced program in the world for treating inherited metabolic storage diseases such as ALD. Our program gives hope to patients and their families dealing with this disease that damages the brain, nervous system and leads to shut down of the body. Today patients are living longer, better-quality lives than ever expected.


  • World leaders in blood and marrow stem cell clinical care, continuing our legacy as home of the first successful bone marrow transplant in 1968
  • Highly experienced in this relatively new form of treatment, having performed more than 5,500 blood and marrow stem cell transplants to date
  • Multidisciplinary care from specialists in endocrinology, genetics, neurology, neuropsychology, neuroradiology, and physical and occupational therapy— all experienced in ALD
  • Related specialty care in endocrinology to treat Addison’s disease (often associated with ALD) includes routine MRIs, physical and occupational therapy, genetic counseling and long-term follow-up care



  • Successfully pioneered the use of the drug N-acetylcysteine (in 2005) in patients with advanced cerebral ALD, slowing the diseases’ rapid progression as patients go through transplantation
  • Developing new treatments for ALD (and the only center studying new techniques with HCT for advanced ALD) through exploration of basic biology of metabolic storage diseases, use of reduced intensity preparative regiments to make transplantation safer, minimizing transplant related complications and addressing late effects of BMT
  • Collaborating with researchers round the world in exploring new and safer ways to treat ALD and improve the quality of life for those with the disease
  • Research studies and clinical trials available for patients


Central Scheduling: 612.672.7422

Provider Referrals: 612.672.7000

Administrative Offices: 612.884.0600

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