Her Brother Participated in Trials for Vital Spinal Muscular Atrophy Drug, and Now She’s Denied Treatment Access
<a href="https://pixabay.com/users/multifacetedgirl/">multifacetedgirl</a> / Pixabay

Her Brother Participated in Trials for Vital Spinal Muscular Atrophy Drug, and Now She’s Denied Treatment Access

According to a story from mirror.co.uk, a five-month-old girl named Maryam Malji was diagnosed with spinal muscular atrophy, a progressive, degenerative disease that is ultimately fatal without treatment. Her brother…

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Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial
<a href="https://pixabay.com/users/DarkoStojanovic/">DarkoStojanovic</a> / Pixabay

Possible Treatment for Niemann-Pick Disease Type C Performs Well in Clinical Trial

According to a story from labiotech.eu, the biotechnology company Orphazyme has been developing an experimental therapy for Niemann-Pick disease type C. In a recent Phase 2/3 clinical trial, the investigational…

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