Charcot-Marie-Tooth Disease Clinical Trial
Official title:
Hearing and Balance Disorders in Peripheral Neuropathy
The long term goal of this proposal is to precisely characterize the auditory and vestibular abilities of children with Charcot-Marie-Tooth (CMT) and how these abilities evolve during the progression of the disorder. This information will be used to refine the management methods for hearing loss and vestibular disorders in these patients. Given that the phenotypic severity is variable within the CMT patient population, we predict that not all CMT patients will present with auditory and vestibular dysfunction. We will therefore collect specimens (i.e., buccal swabs and saliva) from study participants so that their DNA can be isolated and used to determine the genetic basis for auditory and vestibular dysfunction in peripheral neuropathies.
Aim #1: Evaluate the prevalence of auditory and vestibular disorders in children diagnosed with Charcot-Marie-Tooth. We hypothesize that a significant number of children with Charcot-Marie-Tooth (CMT) will develop auditory and vestibular neuropathies, due to the progression of the disorder. We will examine the characteristics of auditory and vestibular abilities of children and how these relate to the type of CMT. Aim #2: Examine the progression of hearing and vestibular abilities during the course of the disorder. We hypothesize that the hearing and vestibular abilities of some children with CMT will deteriorate with time. We will repeat the auditory and vestibular testing on a yearly basis to precisely describe the progression of these abilities. ;
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