Researchers Probe Hearing Loss from Niemann-Pick C Disease Researchers from the National Institutes of Health have partnered with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C—which contributes to significant and progressive hearing loss—and other lysosomal storage disorders. Audiologists and other research staff from the NIH’s National Institute on Deafness and Other ... News in Brief
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News in Brief  |   March 01, 2015
Researchers Probe Hearing Loss from Niemann-Pick C Disease
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Hearing Disorders / Special Populations / Genetic & Congenital Disorders / News in Brief
News in Brief   |   March 01, 2015
Researchers Probe Hearing Loss from Niemann-Pick C Disease
The ASHA Leader, March 2015, Vol. 20, 11. doi:10.1044/leader.NIB2.20032015.11
The ASHA Leader, March 2015, Vol. 20, 11. doi:10.1044/leader.NIB2.20032015.11
Researchers from the National Institutes of Health have partnered with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C—which contributes to significant and progressive hearing loss—and other lysosomal storage disorders.
Audiologists and other research staff from the NIH’s National Institute on Deafness and Other Communication Disorders are participating in a phase I clinical trial of cyclodextrin, led by the National Institute of Child Health and Human Development.
Lysosomal storage disorders primarily affect children, and some, including NPC, may be fatal by early adulthood. Fatty materials in the cells and tissues of the body accumulate, damaging the brain, peripheral nervous system, liver, and other organs and tissues. People with NPC may develop difficulty swallowing and walking, and may progressively lose their vision and hearing.
There is no effective treatment for NPC, but NIH researchers have developed cyclodextrin as a potential therapy. Cyclodextrin, however, has been shown to contribute to hearing loss. During the phase I clinical trial, NIDCD research audiologists are closely monitoring the hearing of study volunteers to determine what doses of cyclodextrin are dangerous for hearing and who may be most at risk for hearing loss.
NIDCD researchers have also conducted studies in mice with NPC to better understand the role of cholesterol in the auditory system and the mechanisms involved in hearing loss linked to NPC.
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March 2015
Volume 20, Issue 3