For generations, economists, pundits, and holders of public office have told the poor that pulling themselves out of poverty was just a matter of acquiring the right skills, making the…
A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…
APR Applied Pharma Research is a small pharmaceutical company in Switzerland concerned with innovations for rare diseases. They have recently been granted the Orphan Drug designation by the FDA, meaning…
According to a story from Financial Buzz, the biotechnology company Inflazome recently announced that it has completed a phase 1 clinical trial. This trial was testing the company's experimental treatment…
Relapsing polychondritis is a rare disease which causes damage to the cartilage, trachea, and lungs. It's an autoimmune condition meaning an overzealous immune response causes the body to attack its own…
A recent study, conducted my Melinda Kavanaugh from the University of Wisconsin, Milwaukee, examined the role of children who are acting as caregivers for those diagnosed with amyotrophic lateral sclerosis…
According to a recent article published in Pharmaceutical-Technology, uniQure’s gene therapy AMT-130 may lower levels of the huntingtin protein that causes the disorder. An abnormal gene is responsible for damage…
A recent Biospace article covers a news release from 4D Pharma in Leeds, England. 4D has announced the first worldwide clinical confirmation of a biotherapeutic product. MRx0518 has been combined…
TARGET PharmaSolutions Inc. recently announced enrollment of the first patients in its ten year, ten thousand patient asthma study. Professor Michael Wechsler, M.D. pulmonologist at the National Jewish Health organization…
As originally reported in Porphyria News, rare diseases have historically been neglected by traditional medicine; their obscure and often genetic nature made them lack priority, stuck behind a long list…
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