The National Organization for Rare Disorders (NORD) is the largest nonprofit independent organization for rare disease information, research, and advocacy. The organization is devoted to the treatment of all rare…
1. Rare Neuromuscular Disease Primary periodic paralysis (PPP) is group of rare neuromuscular diseases characterized by disrupted communication between nerves cells and skeletal muscles. PPP is a result of failures…
After over a decade, Debbie Zelman, founder of Debbie's Dream Foundation, succumbed to her stomach cancer at age fifty. From the beginning, Debbie's prognosis was not good. When the cancer…
According to The Pharma Letter, over the last 20 years progress with the development of orphan drugs for rare diseases has been made, but many question, is it enough? There…
On Christmas Day, Chris Bombardier, a man suffering from hemophilia B, traveled to Antarctica. While most of the people that go there scientists, Chris has a completely different goal in…
Daniel Downing received a personal visit from certain alien robots from outer space. The Transformers Autobots came to town to hang out with the 5-year-old terminally ill boy who has…
Originally reported by The Straits Times, the Chew family from Singapore does everything they can to keep their nine-year-old daughter alive, even if it means spending their life savings and…
According to a story on PRNewswire, the biopharmaceutical company, Enzyvant, kicked off their Farber Disease Natural History Study in October. Acid ceramidase deficiency, also known as Farber disease, is an obscure…
An international study conducted by researchers at the University of Copenhagen has discovered an essential mechanism that controls wound healing in the intestine. The new discovery shows that the cells…
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