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Intrathecal Therapy May Slow Fatal Niemann-Pick Disease

13:10 EDT 22 Aug 2017 | Medscape

Intrathecal hydroxyproyl-beta-cyclodextrin appears to slow progression of cognitive and speech deficits. Hearing loss was a notable adverse event but was 'manageable' in this devastating disease, say investigators.
Medscape Medical News

Original Article: Intrathecal Therapy May Slow Fatal Niemann-Pick Disease

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