Happy October, everyone! We hope everyone's enjoying this new ~fall weather~ and the endless supply of pumpkin spice flavored food products. This week, we're highlighting four FDA announcements we think…
A diagnosis of Cri du Chat (or 5P-), used to mean institutionalization. The name is french meaning "cry of a cat" which comes from the distinct sound children with the illness…
The United States Food and Drug Administration has approved Copiktra™ (duvelisib). The drug is now approved as a treatment for adult patients diagnosed with refractory or relapsed chronic lymphocytic leukaemia…
How it all started Joy Milne is a retired nurse who helped diagnose her husband with Parkinson's disease. 12 years before the official diagnosis, Joy noticed a change in her…
According to a story from wtvbam.com, the drug development company Alnylam Pharmaceuticals has recently stated that it is looking less likely that its gene silencing therapy givosiran will be getting…
According to a story from BioSpace, the biopharmaceutical company Orphazyme recently announced the results from its Phase 2/3 clinical trial of its investigational drug arimoclomol. This therapy is in development…
Over the weekend, the New York Times ran an opinion piece that caught our attention. "He Got Schizophrenia. He Got Cancer. And Then He Got Cured." Click here to read…
An art exhibit held at the Galerie Gora in Montreal, Canada, has used photographs and short stories to share the experiences of eleven people with acromegaly. You can read the…
According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug…
Happy Friday, everyone! We're trying something new this week. Instead of wrapping up with an Editor's Choice article, we're highlighting four FDA announcements we think you should know. Check them…
According to a story from BioSpace, the biotechnology company BioElectron recently announced that the results from its Phase 2a clinical trial of its experimental product EPI-589 are looking positive. EPI-589…
Just Searching for Answers When diagnosed with a rare disease, cancer, or other illness, it makes sense that you’d research every type of treatment on the market. However, there are…
According to a story from Angioedema News, an investigational product from BioCryst Pharmaceuticals called BCX7353 was able to quickly subdue swelling attacks in hereditary angioedema in clinical trials. In addition,…
According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration…
According to a story from lupus.org, a recent spending package was passed in the US House of Representatives that includes $14 million in funding specifically for researching lupus, an autoimmune…
According to a story from statnews.com, the drug developer Sarepta has recently announced the its clinical trial testing an experimental gene therapy has resumed after the FDA lifted the hold.…
According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…
According to a story from Business Wire, the biotechnology company Abivax recently released the results from its Phase 2a clinical trial. This trial was testing its investigational product ABX464 as…
According to a story from Benzinga, Olivia Goodreau of Colorado is the 14 year old creator of the TickTracker app. The application was recognized by the Department of Health and…
The political party the Scottish Liberal Democrats has voted in support of a motion about myalgic encephalomyelitis (ME). This motion calls for several changes, including stopping graded exercise therapy…
According to a report by the Irish Examiner, one in three people spend at least 45 hours or more caring for a someone living with cancer. Despite this being a…
According to a story from pm360online.com, the global biotechnology company Shire plc recently announced its product Takhzyro (injectable lanadelumab) was approved by Health Canada after priority review. Takhzyro is indicated…
Happy Friday! As the week comes to a close, we want to spotlight four articles. We have advice from a patient with CVID who learned how to manage her condition…
A bill (H.R. 6801) has been introduced that is designed to support the development of rare disease therapeutics. Known as the Rare Disease Fund Act, it was introduced by Congressmen…
According to a story from news-medical.net, the new Evergreen Life health and medicines app could be a major benefit for patients across the UK, particularly patients with rare, chronic diseases…
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