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 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…
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…
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…
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…
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 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…
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According to a recent article in Yahoo News, Johan’s mother’s seven-year-old brother died years ago of chronic granulomatous disease (CGD). The mother, Maren, was told that she also had…
Hemophilia A is the most common type of hemophilia, accounting for 8 of every 10 cases. Treatment in the past consisted of the administration of concentrated factor VIII through…
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Ty Eschenbaum’s world of high school football and working on his family’s ranch and farm changed suddenly after his freshman year when he was diagnosed with leukemia. Ty, who…
The 2019 European Academy of Dermatology and Venereology Congress was held in Madrid, Spain. During this conference, a symposium was held discussing IL-23 inhibition in psoriasis. Here are some of…
According to a story from outsourcing-pharma.com, the US Food and Drug Administration (FDA) has recently started a new initiative with the opening of its Compounding Quality Center of Excellence. Janet…
Orphazyme, a biopharmaceutical company, has recently released the data from their study of arimoclomol. This product is meant for the treatment of Niemann-Pick disease type C (NPC). Orphazyme specializes…
According to an article in BioSpace, the FDA recently approved the PARP inhibitor Lynparza that was developed by Merck and AstraZeneca to be used as maintenance therapy for pancreatic…
According to a story from Medgadget, a team of scientists from Columbia University have been working on a robotic device that could assist patients with spinal cord injuries and impairments…
Doctor Daniel Kastner, director of NHGRI, recently uncovered a new rare disease along with his research team. It’s called CRIA syndrome. This discovery has been published in Nature. CRIA CRIA…
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An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…
The human spirit can be a remarkable force. Robb Freed cycled thousands of miles by himself to raise awareness for epidermolysis bullosa (EB) -- a rare illness that took the…
Treatment for ulcerative colitis differs based on the individual. Where the inflammation occurs, the severity, and the preference of the patient can all influence the treatment. For those with moderate…
TrialJectory is an organization working to match bladder, breast, colorectal, melanoma, myelodysplastic syndromes (MDS), and other cancer patients with the clinical trial that is right for them. They have successfully raised…
GlaxoSmithKline (GSK) has recently completed and released the results of Phase 3 of its study of benlysta. This drug is made for the treatment of lupus nephritis. Their BLISS-LN…
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