Nonpharmacologic Approaches to Spasticity Management

Spasticity, defined as a velocity-dependent increase in muscle tone caused by the increased excitability of the muscle stretch reflex, is a challenging symptom that frequently accompanies severe neurological conditions including stroke, multiple sclerosis (MS), cerebral palsy (CP), and spinal cord injury.1 The mechanisms of spasticity are complex, involving an inhibitory/excitatory disruption to the spinal network…

Current and Future Pharmacological Treatment Strategies in X-Linked Adrenoleukodystrophy

Abstract DMutations in the ABCD1 gene cause the clinical spectrum of the neurometabolic disorder X-linked adrenoleukodystrophy/adrenomyeloneuropathy (X-ALD/AMN). Currently, the most efficient therapeutic opportunity for patients with the cerebral form of X-ALD is hematopoietic stem cell transplantation and possibly gene therapy of autologous hematopoietic stem cells. Both treatments, however, are only accessible to a subset of…

N-acetyl-L-cysteine improves outcome of advanced cerebral adrenoleukodystrophy

Abstract Hematopoietic stem cell transplantation as a treatment for childhood cerebral adrenoleukodystrophy (ALD) has historically only been successful in early disease. As ALD is associated with oxidative damage, we reasoned that adjunctive therapy with an antioxidant agent, N-acetyl-L-cysteine (NAC), may provide protection from rapid neurologic decline in boys with advanced cerebral disease. We report three…