The holiday season is upon us, and nothing says holiday cheer more than the smooth and sultry sounds of Michael Bublé singing Christmas tunes. In fact, Michael Bublé has been promoting…
Most patients diagnosed with Homocystinuria (HCU), a rare metabolic disorder, don't receive their diagnosis until later in life. Scientists were at a loss for why patients with phenylketonuria (PKU), a similar…
According to a story from globenewswire.com, the biopharmaceutical company Marinus Pharmaceuticals, Inc., recently announced the presentation of Phase 2 clinical trial of the drug ganaxolone as a treatment for CDKL5…
According to a publication from FierceBiotech, biotechnology companies SmartZyme BioPharma and Hemoshear Therapeutics have started a joint venture together called Carnot BioSciences. Carnot hopes to use non-conventional protein-based therapy to…
Press releases earlier this year on biotech company, Synlogic, announced quite favorable results on both pre-clinical and clinical data on a unique application of synthetic biology for the rare disease, phenylketonuria (PKU). In just…
If you haven't heard of neurofibromatosis (NF), you’re in surprisingly good company. For most of her life, Rachel Mindrup, an artist, illustrator, and Resident Assistant Professor at Creighton University, who…
Just Like You Dolls Just Like You Dolls is a family owned business based in Australia. They design dolls to match their child owner as closely as possible. This includes…
Earlier this month marked a solemn 34 years since the horrendous Bhopal disaster. The worst industrial disaster in human history killed over 3,800 almost instantly, and tens of thousands in…
This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…
"This is what I have to work with. I’m going to make the best out of it.” That's Madeline Schott's motto. The 18 year old was diagnosed with Spinal Muscular Atrophy…
Phil Pavone owns a pawn shop in Norwich, Connecticut. It's called AZ Pawn. Now, in addition to being a businessman, he's practically created his own nonprofit. It all started one…
According to a story from biotech-finances.com, the biopharmaceutical company miRagen Therapeutics, Inc., recently announced the release of data from a Phase 1 clinical trial of miR-155 inhibitor cobomarsen as a…
According to an article published on BioPortfolio, earlier this week, biotechnology company Vybion Incorporated secured orphan drug designation for its highly experimental Huntington's disease drug INT41. About Huntington's Disease Huntington's…
According to a story from ctvnews.ca, Paul Fitzpatrick of Ontario has been enduring severe eye pain following laser eye surgery for over twenty years before he committed suicide. The man's…
According to DublinLive, a team of scientists at the Royal College of Surgeons in Ireland has this week been awarded €200,000 by the research charity Breast Cancer Now, in order…
It was in 1993, just 25 years ago, that congress passed an act to require more diversity in clinical trials. Specifically, it required more female and ethnic minority representation. Since…
According to a story from PR Newswire, the 60th Annual Meeting of the American Society of Hematology has been generating a lot of press lately as many groundbreaking studies, research,…
This weekend Patient Worthy attended RAREfest, an event held in the UK by the Cambridge Rare Disease Network. It brings together patients and their families, researchers, doctors, and the public to…
INSYS Therapeutics has just released data from their long-term safety study of CBD as a treatment for refractory pediatric epilepsy. The results were positive and we're excited about the future…
This weekend Patient Worthy attended RAREfest, an event being held in the UK by the Cambridge Rare Disease Network to raise awareness and promote discussion around rare disease issues. The…
According to a story from globenewswire.com, the biotechnology company Innate Pharma SA recently announced the latest data from a Phase 1 clinical trial of the company's investigational medical product IPH4102.…
Richard Holl of Transient Global Amnesia (TGA) Project 2018 has played a role in the development of a resource website for patients that have experienced transient global amnesia. The website…
According to a story from Market Screener, an analysis of real-world data (data gathered outside of the clinical setting) suggests that the drug eltrombopag, marketed as Revolade, offers substantial advantages…
According to an article published by Biospace, X4 Pharmaceuticals, based in Cambridge, Massachusetts, has recently announced successful phase two results in clinical trials for their experimental drug X4P-001. The drug…
Patients living with any of the Ehlers-Danlos syndromes (EDS) might be tempted to one day visit a chiropractor. The implications of the condition might also make make them equally hesitant…
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