Oliver Sacks is beloved for his best-selling books that detail case studies of rare neurological disorders. Sacks created a niche when he combined his work as a neurologist with whimsical…
Since April 2019, I have been actively seeking out adults (18+) who are willing to publicly share their stories about their lives with rare and chronic diseases. I feel it…
According to a story from BioSpace, the US Food and Drug Administration (FDA) has recently approved avapritinib (marketed as AYVAKIT) as a treatment for gastrointestinal stromal tumors in cases when…
According to an article from the JAMA Network, the role of the human microbiome in our health is just beginning to be understood, and the manipulation of the human gut…
Sandy Patience is a 57-year-old man from Inverness, Scotland, and he has Huntington's disease. His mother and sister also have this disease, so he has seen others who are affected…
According to a story from euronews.com, the drug maker Novartis has announced that it will give away its breakthrough spinal muscular atrophy gene therapy drug Zolgensma to 100 patients each…
According to a story from OMRF, things were looking dire for Tonja Martin after she was diagnosed with multiple sclerosis. This happened to her in 2010, and she promptly began…
Happy Thursday! We're back with a batch of new article! This week, we have another article from PW contributor, Tom Seaman, who uses his experience with dystonia to help people…
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The biopharmaceutical company Cellectar Biosciences recently announced that the FDA granted its lead phospholipid drug conjugate, CLR 131, Orphan Drug Designation(ODD) for treatment of lymphoplasmacytic lymphoma. ODD is granted by…
AI Therapeutics is a biopharmaceutical company that has created an artificial intelligence platform that is meant to match drugs to new indications. This platform, called Guardian Angel, has matched…
According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company's investigational drug…
Gene therapy has become a technological golden snitch: the end of an era for many genetic disorders if we can grasp the treatment’s potential. However, the technology comes with a…
According to a story from News Amed, Rob Burrow gained fame as a star player for the Leeds Rhinos, a professional rugby team. After a distinguished career, Rob retired from…
Hyperphagia is an extreme hunger and obsession with food, and it is a symptom of Prader-Willi syndrome. While many of the other effects of this condition have treatments, this…
January 24th is Moebius Syndrome Awareness Day! Let's take a deeper dive into what Moebius Syndrome is and what we can do to push awareness on this day (and all…
According to a story from Medscape, a recent study published in the medical journal Pituitary has revealed a significant disconnect between acromegaly patients and their physicians. The study suggests that doctors…
Olmsted syndrome is a rare disease most frequently caused by mutations in TRPV3. For some patients, it is caused by MBTPS2 mutations. It is characterized by palmoplantar keratoderma (PPK) as…
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The American Journal of Managed Care (AJMC) published excerpts from a presentation by Dr. Andrew Wei at the 61st American Society of Hematology (ASH) Annual Meeting. The article stated…
If you are like me and probably most people, in today’s busy world it is a big challenge to be still and mentally at ease. This is such an important…
A diagnosis is something that is not always easy to attain. Without one, people cannot receive the best treatment for their conditions, miss out on a community of others…
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According to a recent article in Pharma Tutor, researchers at NHGRI, a division of the National Institute of Health, identified the biological cause of a newly-discovered autoinflammatory disease they named…
According to a story from Acrofan, the biotechnology company Clover Pharmaceuticals Inc. has recently announced that the first patient has been dosed in its phase 3 clinical study. This study…
January 26th (the last Sunday of January) is World Leprosy Day! Mostly remembered as a Biblical disease that lead to shunning and societal ostracization, the stigma surrounding leprosy has made…
According to a story from Markets Insider, the biopharmaceutical company Principia Biopharma Inc. has recently issued new updates in regards to the timeline of its phase 3 clinical study. This…
By Rachel Whetstone from In The Cloud Copy Shelly Wheaton began a car sign campaign in September to look for a live kidney donor. She was on dialysis because…
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