Update on the Treatment of Ataxia: Medication and Emerging Therapies
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REVIEW
Update on the Treatment of Ataxia: Medication and Emerging Therapies Susan L. Perlman 1 Accepted: 28 September 2020 # The American Society for Experimental NeuroTherapeutics, Inc. 2020
Abstract While rehabilitation therapies always help patients with ataxia, there are currently no FDA-approved treatments for ataxia. Medications are available to treat symptoms that may complicate an ataxic illness, e.g., tremor, myoclonus, dystonia, and rigidity, which are discussed elsewhere in this volume. Spasticity, pain, fatigue, depression, sleep disturbances, cognitive decline, and bowel and bladder dysfunction, if they occur, all have multiple available drugs and therapies for symptomatic use. There is also an extensive literature on off-label uses of various medications to improve imbalance. The pipeline of emerging therapies for symptomatic and possible disease-modifying management of ataxia gives hope that we will soon see the first of many FDAapproved drugs for ataxic illnesses. Key Words Ataxia . rehabilitation . clinical trials . neurostimulation . gene therapy
Introduction Ataxia, as a diagnosis, displays motor symptoms and signs of staggering gait, imbalance, incoordination, action tremor, slurred speech, trouble swallowing, disequilibrium/dizziness, nystagmus, and double vision, typically caused by cerebellar dysfunction on the basis of genetic, acquired, or degenerative causes [1–4]. While these symptoms can specifically come from the cerebellum itself, the disease process may also involve neural pathways outside the cerebellum, including intrinsic brainstem nuclei (oculomotor and bulbar dysfunction), spinal long tracts (spasticity, sensory changes), and supratentorial pathways (basal ganglia symptoms, frontal subcortical cognitive/mood changes [5]). Changes in special sensory pathways (optic, auditory), peripheral motor and sensory pathways, and autonomic pathways can accompany certain ataxic diagnoses. Involvement of any combination of these symptoms can lead to derangement of bowel and bladder function, altered sleep patterns, fatigue, pain, and mood disturbances.
“Pseudo-ataxia,” when the cerebellum is normal, but gait, balance, and coordination are compromised by other factors, can be seen in conditions of muscle weakness, muscle stiffness (spasticity or rigidity), severe peripheral or posterior column sensory loss, peripheral vestibular dysfunction, visual problems, and orthopedic disease. While the multifactorial presentation of an ataxic patient may seem overwhelmingly complex, utilizing any of the multiple available therapeutic interventions for the individual symptom areas can always help [6]. Stopping or slowing of disease progression, however, for any of the types of ataxia, depends on finding the specific causal mechanisms and the disease-modifying therapies that target them. There are as yet no disease-modifying therapies for genetic or degenerative cerebellar disorders. The treatable ataxias remain the acquired ones (vitamin deficiency, other metabolic disturbances, toxin exposure, medicat
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