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May 2002

Experimental Neurotherapeutics: Leaps and Bounds

Arch Neurol. 2002;59(5):689-691. doi:10.1001/archneur.59.5.689

NEUROLOGY HAS emerged from an immature era of therapeutic nihilism to witness incremental and tangible advances in the treatment of a variety of disorders. These gains have emanated largely from sound scientific underpinnings and form the basis for the rational selection and objective testing of experimental interventions. Neurologic therapeutics is finally reaping benefits from the convergence of laboratory-based investigations and randomized clinical trials.

In the past decade, treatments have emerged for several neuromuscular diseases. Several seemingly esoteric muscle disorders are now known to represent functional disruption of the ion channels in skeletal muscle, leading to more targeted and effective suppression of their paroxysmal manifestations.1 Even the inexorable progression of amyotrophic lateral sclerosis, to death or tracheostomy, has shown evidence of slowing, albeit marginally, with riluzole treatment.2 Migraine therapy has been transformed by a variety of specific serotonin agonists and the emerging insight that cortical spreading depression is involved in the pathogenesis of this paroxysmal disorder.3 What formerly was considered intractable epilepsy has succumbed in carefully selected and monitored patients who have undergone targeted ablative surgery.4 The range of antiepileptic pharmacotherapy has also expanded to include better-tolerated agents that treat specific types of seizures based on their clinical and electroencephalographic signatures. In addition, some new antiepileptic agents effectively treat chronic pain and a host of other ailments.

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