According to a story from Drug Development Technology, the drug developer Translate Bio has recently reported interim results from its phase 1/2 clinical trial. This trial is testing the company's…
Brenda has both psoriatic arthritis and psoriasis. She's lived with the conditions since she was a child, learning to care for herself and deal with the challenges that arose. By…
According to a story from the Scientific American, new research is revealing the potential of the human microbiome as a method for treating health problems. Research is also continuing to…
According to a press release from American biotechnology company Neurogene, the Company has kicked off a sponsorship of Invitae Corporation's "Detect Lysosomal Storage Diseases" program, which will offer genetic testing…
According to a story from Charcot-Marie-Tooth Disease News, Charcot-Marie-Tooth disease is actually somewhat common in the US compared to other rare diseases. In fact, it is estimated that around 150,000…
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Trust is vital when it comes to medical care. People put their most important questions and qualms into the hands of doctors and medical staff and expect they can rely…
August 3rd is CLOVES Syndrome Awareness Day! The CLOVES Syndrome Community (CSC) is an organization that "supports, educates, empowers and improves the lives of those affected by CLOVES Syndrome" and…
According to a story from PR Newswire, the biopharmaceutical company Biohaven Pharmaceutical Holding Company, Ltd. has announced that the first patient has signed up for the company's phase 3 clinical…
According to a story from home.suddenlink.net, the biopharmaceutical company X4 Pharmaceuticals recently announced that the European Commission has granted Orphan Drug designation to the company's lead investigational drug candidate, mavorixafor.…
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 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 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…
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…
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.…
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 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…
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…
According to a story from globenewswire.com, the biopharmaceutical company Mustang Bio, Inc. has recently announced that the US Food and Drug Administration (FDA) has recently awarded the company Orphan Drug…
According to a story from drugs.com, the pharmaceutical company Pfizer, Inc. recently issued an announcement detailing the approval of its drug Ruxience (rituximab-pvvr), a biosimilar to the widely used monoclonal…
According to a press release from the Kentucky-based Apellis Pharmaceuticals, the Company has initiated a program to investigate the use of APL-9, an investigational drug, in gene therapies that involve…
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