News & Events

In this 2017 interview, Paul Orchard, MD, Professor in the Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, answered questions about adrenoleukodystrophy (ALD) and the services M Health Fairview has to offer for care and treatment of this condition. Through research and innovation, Dr. Orchard and the rest of the interdisciplinary team at M Health Fairview Masonic Children's Hospital work on finding and innovating alternatives to current treatments to increase treatment success. 

In 2013, shortly after Brock Groh was born, he was diagnosed with adrenoleukodystrophy (ALD), a rare genetic disorder that later developed into cerebral ALD, a life-threatening form of the disease. Brock received a life-saving bone marrow transplant at M Health Fairview Masonic Children's Hospital. To express their appreciate for the care their son received while at M Health Fairview, Brock's family started a nonprofit, X out ALD, to donate in support of research at the hospital facility. 

In this 2019 interview, Troy Lund, MD, PhD, Professor and Fellowship Program Director in the Division of Pediatric Blood and marrow Transplantation & Cellular Therapy at the University of Minnesota, discussed rare diseases as a way to promote awareness in support of Rare Disease Day. As of 2019, there are approximately 7,000 rare diseases, affecting around 25 to 30 million Americans. While treatments for rare diseases are advancing, many conditions still lack effective treatment options.

In 2022, the Food and Drug Administration (FDA) approved a gene therapy for cerebral adrenoleukodystrophy (ALD), a rapidly progressing rare genetic neurodegenerative disease that impairs the body's ability to break down very long-chain fatty acids. This treatment, developed by a team of physicians and researchers at M Health Fairview Masonic Children’s Hospital—a nationally recognized rare disease specialty center—replaces the defective gene responsible for ALD with a functional copy. This breakthrough provides a vital treatment option for a rare condition that affects approximately one in 17,000 people

In 2021, siblings Grace and Grant Goodin received the first bone marrow transplants for Multiple Sulfatase Deficiency (MSD) at M Health Fairview Masonic Children’s Hospital. This groundbreaking treatment was the first of its kind globally, led by a team of pediatric physicians and rare disease specialists. MSD is an exceptionally rare and often fatal genetic disorder, with only about 100 cases ever diagnosed worldwide, including Grace and Grant.