According to a story from People Magazine, parents Deborah and Antoine Vauclare were devastated when they found out that their son Léo was born with an incredibly rare genetic disorder called infantile…
We need to talk about adult rare disease patients. Adults who have a rare disease diagnosis are faced with a blaring issue-- I'm speaking for a large number of adults here,…
According to a story from BioPortfolio, the biopharmaceutical company AlzProtect recently announced that PAREXEL Biotech, which is a subsidiary of PAREXEL International Corporation, will be expected to conduct a Phase…
Happy Wednesday! Today, we're highlighting an article about gene therapy for MPS, followed by a story about a singer with Achalasia. After that, we have an article about app for…
I have lived with pain almost my entire life. Prior to the age of 30, when I developed chronic pain from a neurological movement disorder called dystonia, I experienced pain primarily…
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…
According to a story from the Tenterfield Star, a fundraiser was recently held for Tommy Johnston, a one year old boy who was diagnosed with the rare Niemann-Pick disease type…
According to a story from globenewswire.com, the biopharmaceutical company Xenon Pharmaceuticals, Inc., recently announced the latest updates on the progress of two of its experimental products. The first is called…
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