Pediatric Blood & Marrow Transplantation Center
We're here to help: (888) 601-0787 or (612) 273-2800

Building on a Legacy

Building on a Legacy with a Vision for the Future.

While newborn screening programs vary from state to state, many are expanding their screening programs to test for diseases such as ALD and Hurler syndrome. Learning that their newborn has tested positive for a rare disease is devastating and confusing for parents. The Inherited Metabolic Disorder Comprehensive Care Program at University of Minnesota Masonic Children’s Hospital is available to these families, as well as to families with a child diagnosed later in the disease process. We can provide answers to many questions: What do we do now? How should we prepare? Does my child need immediate intervention? What do we need to be concerned about, and what warning signs should I look for? Our team of professionals including BMT physicians, neurologists, neuropsychologists, endocrinologists, cardiologists, orthopaedic specialists, ophthalmologists, ENT specialists, genetic counselors and physical therapists works with families to develop the best plan to meet the needs of every child.

Some metabolic diseases may require immediate medical intervention from the onset of diagnosis. Others, such as ALD without adrenal insufficiency or cerebral white matter changes, don’t require intervention until indicated by medical testing. But it may be possible to identify which patients are at risk for cerebral disease and prevent brain dysfunction before it begins. Gerald Raymond, MD is focusing his research on early interventions to prevent the onset of cognitive decline in boys with ALD. Dr. Raymond was instrumental in the use of the therapeutic remedy, Lorenzo’s oil, in the United States and is spearheading projects to develop alternative agents to treat early stage ALD.

Not all people living with inherited metabolic diseases are children. Occasionally disease symptoms first manifest in early adulthood. Some of these patients could benefit from blood or marrow transplantation (BMT), but more needs to be learned about this unique patient population. University of Minnesota researchers are working to develop better treatment options to meet the needs of adult patients with metabolic disorders.

Patients with advanced disease, particularly those with advanced neurologic symptoms, have historically been difficult to treat. Because BMT has not been shown to be helpful for these patients, new approaches are needed. The University of Minnesota is working to develop new options for patients with advanced metabolic disease with or without transplantation. This may include the administration of other cell types or drugs that have not yet been tested for these diseases.

Our comprehensive care team, including world-renowned specialists in BMT -- neurologists, endocrinologists, neuropsychologists, cardiologists and orthopaedic specialists -- is available for assessment and consultation. The team works together to create an individualized monitoring and treatment plan for each patient.

To learn more about the Inherited Metabolic Disorders Comprehensive Care Program at University of Minnesota Masonic Children’s Hospital, contact Paul Orchard, MD, Weston Miller, MD, Troy Lund, MD, or call Patty Kleinke with the BMT Program office at 612-273-0857 or 888-601-0787 (toll free).

Journey Clinic - Pediatric Blood and Marrow Transplantation Center

2450 Riverside Avenue
Minneapolis, MN 55454
612-365-1000
612-365-1000 TTY: 612-672-7300

For information about the Adult Blood and Marrow Transplantation program, click here:

We're Here To Help

We have been providing innovative care to children in need of a blood and bone marrow transplant for over 40 years. Call us, we're here to help.

(888) 601-0787 or (612) 273-2800

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