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As set forth in a recent MD Anderson Cancer Center publication, targeted therapy makes it possible to personalize cancer treatment. It is also known as “precision medicine”. For about forty…
According to a story from Cancer Health, a team of scientists affiliated with Mayo Clinic, UC San Francisco, and the Chan Zuckerburg Biohub have discovered a new rare, autoimmune disease…
According to a story from Financial Buzz, the biopharmaceutical company Soleno Therapeutics recently provided an update on the progress of the Company's Phase 3 clinical trial. This trial is evaluating…
According to a publication from the Rheumatologist, past studies have suggested that type 1 interferons may play a significant role in determining susceptibility to several classic connective tissue diseases. Although…
According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics, Inc., has recently announced that the European Commission has given the company's investigational drug candidate mirdametinib Orphan Drug designation.…
According to a story from BioSpace, the biopharmaceutical company CANBridge Pharmaceuticals announced that it has recently submitted a New Drug Application with China's National Medicinal Products Administration (NMPA) for its…
According to a story from Gaucher Disease News, the German drug company Centogene has recently released a free smartphone app called MyLSD which will allow patients with Gaucher disease monitor…
According to a story from Korea Biomedical Review, two Korean drama television shows, Partners for Justice Season 2 and Doctor John have recently depicted Fabry disease as key components during their run, which…
Happy Thursday! This week, we're highlighting a PW contributor's story exploring blepharospasm and mental health and an article on two sisters with a rare connection. Following that, we have a…
According to a story from Markets Insider, the biopharmaceutical company GENFIT recently announced that both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have awarded…
According to a press release from the Danish biopharmaceutical company Orphazyme, the Company has filled target enrollment for its phase 3 clinical study of arimoclomol in patients with amyotrophic lateral…
I had the pleasure of attending the Glut1 Deficiency Foundation’s 2019 Conference. While this article can't possibly do the event justice and encompass all it had to offer, we will…
According to a story from Xconomy, the US Food and Drug Administration (FDA) made a controversial decision three years ago when it approved the drug eteplirsen as a treatment for…
According to a press release from American biotechnology company Celgene Corporation, the American Food and Drug Administration (FDA) recently approved the Company's drug Otezla (generic name apremilast) for the treatment…
According to a publication from Fabry Disease News, biotechnology company Avrobio recently announced that early phase 1 and 2 clinical studies of experimental Fabry treatment AVR-RD-01 have yielded encouraging results.…
According to a story from Cushing's Disease News, a recent study has confirmed that repeated transsphenoidal surgery can be effective in treating Cushing's disease in patients that have experienced remission…
According to a story from Healio, the US Food and Drug Administration's (FDA) Arthritis Advisory Committee recently voted 10-7 in favor of recommending the drug nintedanib as a treatment for…
The struggle of living with a rare disease and dealing with insurance issues was in the spotlight on national television. During Tuesday night's 2020 Democratic debate for the president, Senator…
According to a story from Angioedema News, a recent clinical trial has demonstrated that the drug conestat alfa (marketed as Ruconest) was able to successfully treat 96 percent of acute…
According to a story from the Broad Institute, there are still many mysteries to uncover related to inflammatory bowel diseases, which includes the rare form ulcerative colitis. The understanding of…
A Colorado mom is on a mission to raise money for her daughter's type 1 spinal muscular atrophy (SMA) treatment -- that would save her child’s life. Let's hear little Maisie…
According to a story from Angioedema News, a team of researchers has used data from a recent study to provide up to date recommendations for diagnosing hereditary angioedema in the…
An article from Comic Sands recently covered the story of a UK mother, Alison Beesley, who is now committed to taking care of her daughters Eliza and Eleanor full time. Eliza is nine years old and…
Talking about mental illness is never easy but I wanted to share my experience of it with you. In my case, it came as part of my struggle with Benign…
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Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…
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