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Fernandes RJ, Hirohata S, Engle JM, et al

In the future, spinal cord targeting needs to be enhanced by improving drug delivery systems (such as nanocarriers or targeted modifications), optimizing dosage strategies in combination with pharmacokinetic-pharmacodynamics models, and promoting multi-stage clinical trials to clarify its clinical translational value

Salim A, Leman JA, McColl JH, Chapman R, Morton CA

Adrenal insufficiency is monitored closely and is treatable with hormonal supplementation, neurological aspects of X-ALD are far more challenging to treat