According to a story from fda.gov, the US Food and Drug Administration (FDA) has approved the drug pexidartinib (marketed as Turalio) as a treatment for tenosynovial giant cell tumor (TGCT)…
According to a story from First World Pharma, the biopharmaceutical company Mustang Bio, Inc. recently announced that the US Food and Drug Administration (FDA) has approved their investigational new drug…
According to a story from Pharmaceutical Technology, the company Stoke Therapeutics experimental drug candidate, known as STK-001, has earned Orphan Drug designation from the US Food and Drug Administration (FDA).…
RAREsummit19 is a 1 day summit focusing on patient-centricity in rare disease progress. What if we were able to share information with the power to revolutionise the current rare…
Happy Thursday! This week, we're highlighting an story about a woman with Bechet disease who advocates for herself and others. Next, we have an article discussing the difficulty of caring…
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 True Viral News, anyone who has tried to get something done through governmental means are probably familiar with getting rebuffed and redirected at every turn.…
According to a story from globenewswire.com, the drug company Amicus Therapeutics recently announced that Argentina's Agency of Medicines, Food, and Medical Devices (ANMAT) has granted approval for the drug migalastat…
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 press release from British biotechnology company Immunocore, the Company has partnered with Pulse Infoframe (a Canadian data analytics company) to support the development of an international database…
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 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 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 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 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…
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…
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…
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