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…
According to a story from sectorpublishingintelligence.co.uk, the clinical stage pharmaceutical company Retrotope recently announced that the first patient had been dosed in its Phase 2/3 clinical trial. The trial will…
Pantothenate kinase-associated neurodegeneration (PKAN) is a neurodegenerative disorder caused by a mutated PANK2 gene. This mutation disrupts a metabolic pathway, decreasing the body's levels of CoA. CoA, or coenzyme A,…
This is an interview I did with Scott Davidson who has a podcast called Living Adaptive, where he interviews people that have adapted to significantly difficult situations in their lives.…
Happy Wednesday! As the new month begins, we want to spotlight four articles. First, we have an incredible story about a Cushing's syndrome survivor and advocate and her talented service…
According to a story from MedicalXpress, it was around 30 years ago when two sons of Marsha and Allen Barnett, named Chuckie and Michael, both passed away from Leigh syndrome.…
According to a story from rdmag.com, the biopharmaceutical company IntraBio Inc., recently announced that its IB1000 series of investigational therapeutic compounds have been granted Orphan Medicinal Drug Designation as a…
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