Hereditary Angioedemia (HAE) results when the blood is missing the C1-inhibitor protein. It causes unpredictable and sudden attacks of swelling on various parts of the body. It can affect practically…
Happy Wednesday! With Hanukah wrapped up, and Christmas just around the corner, things are getting pretty crazy. Take a second to relax and check out these four stories. First, we…
According to a story from BioSpace, the cancer drug company Gibson Oncology, LLC has recently announced that the US Food and Drug Administration (FDA) has awarded Rare Pediatric Disease designation…
According to a story from pm360online.com, the gene therapy company Fibrocell Science, Inc., recently received an investment of $900,000 from the Epidermolysis Bullosa Medical Research Foundation (EBMRF) and the EB…
At Patient Worthy, we are dedicated to the covering the latest stories about rare diseases that appear in the news cycle. While this is an important part of our mission…
According to a story from curetoday.com, a recent Phase 2 trial has demonstrated that bortezomib (marketed as Velcade) has the potential to be an effective therapy for patients with mantle…
According to a story from Proactive Investors, the biotechnology company Conatus Pharmaceuticals recently reported that the company's drug Emricasan failed to achieve the primary endpoint in a Phase 2b clinical…
According to a story from the Greenock Telegraph, twelve year old Megan McGarva, who was born with CDLK5 deficiency, will soon have to face a serious spinal operation. She has…
Sarcoidosis is an inflammatory condition which can affect any organ in people of any age. The prognosis ranges from benign to debilitating. Granulomas, or clumps of cells, build up in…
A UCLA-led research team has developed a spray gel embedded with immune-boosting drugs for post-surgery cancer patients to thwart reccurrence and spreading of the disease. After initial testing on animals,…
According to a story from PR Newswire, the Blueprint Medicines Corporation recently announced that latest results from a Phase 1 clinical trial of avapritinib as a treatment for advanced systemic…
Andrew Taylor celebrated his 50th birthday this year. This is a big year for anyone, but especially for Taylor, who was told he would not make it to the…
DrugPatentWatch recently published a copy of a paper originally published in the Journal of Applied Pharmaceutical Science. In the paper, authors Emanuel Almeida Moreira de Oliveira and Karen Luise Lang…
According to a story from pm360online.com, the biopharmaceutical company Ultragenyx Pharmaceutical Inc., in collaboration with Kyowa Hakko Kirin Co. Ltd, and Kyowa Kirin International PLC, recently announced that injectable burosumab…
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…
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…
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 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…
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 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…
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…
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…
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.…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
