According to a story from MD Edge, a phase 2 clinical trial testing the drug concizumab as a treatment for hemophilia A and hemophilia B demonstrated substantial capability in reducing…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is actually a term used to classify a few different conditions. Two of these are Crohn's disease and ulcerative colitis. These are rare…
According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…
Usually when people think of a seizure disorder, they think of something called tonic-clonic seizures or episodes where a person loses consciousness, has muscle stiffness, spasms, and jerky movements.…
According to a story from globenewswire.com, Akcea Therapeutics and Ionis Pharmaceuticals recently announced the release of top line results from a clinical study. This trial tested the drug WAYLIVRA as…
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…
According to a story from Direct Relief, when Yusra was just a year old, she began to have mysterious bouts of fever. Her family had no explanation. By age three,…
According to a publication from ScienceDaily, a new study of propranolol suggests explanations for its ability to shrink certain benign tumors in infants and relieve symptoms of hypotrichosis-lymphedema-telangiectasia syndrome. About…
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…
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