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The Myelin Project was established in 1989 by Augusto and Michaela Odone for their son Lorenzo who suffered from adrenoleukodystrophy (ALD), a rare genetic disease that deteriorates myelin in the brain and central nervous system. Although not medical doctors, the Odones went on an investigative journey into the disease and were able to develop an oil treatment that has proven to slow the progress of the disease – and they named it Lorenzo’s Oil – which has now become an internationally recognized treatment, yet still not approved by the US FDA.  The story of the Odones’ struggle was dramatized in George Miller’s 1992 film Lorenzo’s Oil starring Nick Nolte and Susan Sarandon.

RESEARCH

ADVOCACY

Adrenoleukodystrophy (ALD) is a rare x-linked genetic brain disease that affects roughly 1 in 15,000 people. It has four different forms, or phenotypes. The most devastating appears in childhood, generally between the ages of four and ten years old. Normal, healthy boys suddenly regress rapidly. Symptoms begin with hyperactivity, withdrawal, and/or difficulty concentrating. As the disease continues to destroy myelin in the brain and nervous system, symptoms grow worse including blindness, deafness, seizures, loss of muscle control, and progressive dementia. Often this leads to either death or permanent disability usually within 2 to five years of diagnosis.

Adrenomyeloneuropathy (AMN) is the adult onset of adrenoleukodystrophy (ALD). In about half of the boys who inherit the mutated ALD gene, symptoms of the disease do not develop until young adulthood, and in general, they progress more slowly. Beginning in their 20s and 30s, these young men exhibit neurological based motor lesions in their extremities. These lesions progress over many years and are inevitably accompanied by moderate to severe handicap and in many cases, these symptoms lead to death.