Phenylketonuria Patient Didn’t Receive Life-Altering Treatment for 10 Years, Alludes to Bigger Problem

Within the pharmaceutical industry, we need 1) better policies for patients and 2) improved communication/education of the policies that do exist for patients and the rights that they have. Ultimately,…

Continue Reading Phenylketonuria Patient Didn’t Receive Life-Altering Treatment for 10 Years, Alludes to Bigger Problem

Experimental Treatment for Neuromyelitis Optica Earns Breakthrough Therapy Designation

According to a story from BioSpace, the biotechnology company Viela Bio recently announced that the US Food and Drug Administration (FDA) has awarded an experimental therapy in development by the…

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Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa

The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…

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