Dietary therapy Initial attempts at dietary therapy in<unk>  involved restricting the intake of very-long chain fatty acids (VLCFA). Dietary intake is not the only source for VLCFA in the body, as they are also synthesized endogenously. This dietary restriction did not impact the levels of VLCFA in plasma and other body tissues. After the realization that endogenous synthesis was an important contribution to VLCFA in the body, efforts at dietary therapy shifted to inhibiting these synthetic pathways in the body. The parents of Lorenzo Odone, a boy with<unk> , spearheaded efforts to develop a dietary treatment to slow the progression of the disease. They developed a mixture of unsaturated fatty acids (glycerol trioleate and glyceryl trierucate in a 4:1 ratio), known as Lorenzo's oil that inhibits elongation of saturated fatty acids in the body. Supplementation with Lorenzo's oil has been found to normalize the VLCFA concentrations in the body, although its effectiveness at treating the cerebral manifestations of the disease is still controversial and unproven. Trials with Lorenzo's oil have shown that it does not stop the neurological degradation in symptomatic patients, nor does it improve adrenal function, but asymptomatic patients, and speculatively AMN variants without cerebral involvement, as well as female carriers may benefit from early intake of oleic and erucic acids in addition to VLCFA restriction. Lorenzo's oil A solution of 4 parts glycerol trioleate and 1 part glycerol trierucate, which are the triacylglycerol forms of oleic acid and erucic acid, has been tested for ADL. Results are mixed, showing possible benefit for asymptomatic patients in conjunction with dietary therapy, but little or no benefit for those with symptomatic<unk> . Transplant While dietary therapy has been shown to be effective to normalize the very-long chain fatty acid concentrations in the plasma of individuals with<unk> , allogeneic hematopoietic stem cell transplants is the only treatment that can stop demyelination that is the hallmark of the cerebral forms of the disease. In order to be effective, the transplant must be done at an early stage of the disease; if the demyelination has progressed, transplant can worsen the outcome, and increase the rate of decline. While transplants have been shown to be effective at halting the demyelination process in those presenting with the childhood cerebral form of<unk> , follow-up of these patients has shown that it does not improve adrenal function. Gene therapy For patients where an appropriate match for a transplant cannot be found, there have been investigations into the use of gene therapy. Appropriate vectors are selected and modified to express wild type ABCD1, which is then transplanted into the patients using a similar procedure as for a bone marrow or stem cell transplant. Gene therapy has only been tried on a small number of patients, mainly in France. These patients were only considered for gene therapy after there was no HLA match for a traditional transplant. In two reported cases, the gene therapy was successful, with a resolution of the demyelination process up to two years after the procedure. Although the gene therapy was successful in resolving the neurological symptoms, plasma VLCFA levels remained elevated. Elivaldogene autotemcel is pending authorization by the European Commission . Adrenal insufficiency Treatment of the adrenal insufficiency that can accompany any of the common male phenotypes of<unk>  does not resolve any of the neurological symptoms. Hormone replacement is standard for<unk>  patients demonstrating adrenal insufficiency. Adrenal insufficiency does not resolve with successful transplant; most patients still require hormone replacement.
Adrenoleukodystrophy
Hereditary inclusion body myopathy
Inclusion body myositis
Mitochondrial myopathy
Familial amyloid neuropathy
Familial amyloid polyneuropathy
Charcot–Marie–Tooth disease
Lambert–Eaton myasthenic syndrome