Cystinosis Cystinosis is a rare disease which is caused by a buildup of cystine in the body's cells. This buildup impacts various organs including the brain, eyes, liver, kidneys, and muscles.…
According to a story from Biotecnika, Penn Medicine & Inovio Pharmaceuticals Inc. have recently announced that the US Food and Drug Administration (FDA) has signaled its approval for the first…
A new study published in the journal Cell Reports identifies possible new treatments for absence (silent) seizures in children with Dravet syndrome. Children with Dravet syndrome have developmental delays, cognitive…
Mucolipidosis Type IV Mucolipidosis Type IV (ML-IV) is a rare disease which causes a reduced mental capacity and blindness. ML-IV is caused by a missing or malfunctioning TRPMLI protein. Most patients never…
According to a story from dailymail.co.uk, 23 year old Ruby Hill of New Zealand was recently released from the hospital, but it wasn't because she had been successfully treated. It…
According to a story from kios.org, firefighters across the country tend to get cancer, including many rare cancers like mesothelioma, at significantly greater rates than the general population. This is…
According to a story from Fabry Disease News, the drug developer Avrobio recently announced that the US Food and Drug Administration (FDA) has given the company's experimental drug AVR-RD-01 Orphan…
According to a story from bioengineer.org, it has long been known that people with the rare disorder Duchenne muscular dystrophy are at a greater risk of developing a rare cancer…
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