Adrenoleukodystrophy

(Baltimore, MD) -- Kennedy Krieger Institute will join the National Organization for Rare Disorders (NORD) and others around the world in observing Rare Disease Day on February 29. The purpose is to focus attention on the needs of patients and families affected by rare diseases.

"This is a global observance," said Peter L. Saltonstall, president and CEO of NORD. "Individuals and organizations around the world will all be sharing stories of how rare diseases affect their lives."

The challenges of living with a rare disease, he said, include:

Ramón and Maritza Casso loved their first born son, Juan, with all of their hearts. Like many parents they wanted far more for him than they wanted for themselves. And when their second son, José, arrived seven years later, they felt the same way.

It has been said that a truly extraordinary man is a man who thinks himself completely ordinary. Hugo W. Moser was such a man. A scientist, mentor, advocate, father, husband, friend, he is surrounded by an acclaim that bears the weight of work well done, personal passion, and a nobility of purpose.

(Baltimore, MD) Kennedy Krieger Institute will be joining the National Organization for Rare Disorders (NORD) and others around the world in observing World Rare Disease Day on February 28, 2011. On this day, millions of patients and their families will share their stories to focus a spotlight on rare diseases as an important global public health concern.

The Kennedy Krieger Institute's faculty and staff extend our deepest condolences to the family of Lorenzo Odone, who passed away on Friday, May 30, after surviving adrenoleukodystrophy (ALD) for more than 20 years.

Over the course of Lorenzo's life, his father, Augusto Odone, made innumerable contributions to the quest for answers about his son's rare and devastating disease. His efforts impacted the scientific field in ways that have dramatically changed the fate of those who followed in Lorenzo's footsteps with diagnoses of ALD and other demyelinating disorders.

Baltimore, MD - Hugo W. Moser, M.D., Ph.D., recognized throughout the world for his research on genetic disorders that affect nervous system function in children, passed away on Saturday, January 20th following a courageous battle with cancer. He leaves behind a legacy of advancing the science surrounding some of the most complex genetic disorders, particularly the disease adrenoleukodystrophy (ALD), as profiled in the 1992 release of the motion picture Lorenzo's Oil.

(Baltimore, MD) - Use of Lorenzo's Oil in young boys who have been diagnosed with but are not yet showing signs of a pediatric neurological disorder known as X-linked adrenoleukodystrophy (X-ALD) may prevent the disease from developing in the body. According to a study of male children published today in the Archives of Neurology, use of the oil prevented onset of the disease in a majority of cases.

GHENT, Belgium - Dr. Hugo Moser, director of Neurogenetics at Kennedy Krieger Institute in Baltimore, will present the findings of a major, 10-year study of Lorenzo's Oil' therapy in children with adrenoleukodystrophy (ALD) today at a scientific symposium at Ghent University.