Dana-Farber Cancer Institute has just announced research updates on multiple myeloma and waldenstrom macroglobulinemia which indicate significant progress in the search for treatments for these diseases. The Findings 1) There…
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…
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…
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…
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…
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…
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 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…
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,…
According to a story from BioPortfolio, the global biopharmaceutical company Celgene Corporation recently announced the release of early data from the dose-escalation portion of a Phase 1/2 clinical trial of lisocabtagene…
Chronic myelomonocytic leukemia, or CMML, is a rare condition. However, it has similarities to myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPN). That being said, it still a unique diagnosis, and…
Huntington's Disease is a neurodegenerative condition caused by a genetic mutation. It results in progressive decline of motor skills. It also affects cognitive function. Patients experience decline for 15-20 years…
Sickle cell disease (SCD) is a blood disorder. SCD patients have abnormal hemoglobin, or sickle hemoglobin, caused by a genetic mutation in the beta-chain. It results in deformed and inflexible…
Happy Wednesday! As the new month begins, we want to spotlight four articles. First, we have an incredible story about a Cushing's syndrome survivor and advocate and her talented service…
In the UK, a think tank determines access to rare disease drugs based on their perception of an individual's quality of life. That idea has also been developed in the…
Mantle cell lymphoma is caused by a genetic mutation which produces cancerous white blood cells. Of course, these are the cells which fight infection in a normal body. Therefore in…
According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…
According to a story from The San Diego Union-Tribune, the Pancreatic Cancer Prevention and Screening Clinic at UC San Diego Moores Cancer center has opened for the first time this…
When Deborah, a New Yorker, flew over to Paris to do an internship for an architecture office, she never knew she would be flying across the world and meeting…
According to a story from BioPortfolio, the gene therapy company Rocket Pharmaceuticals has recently announced that the company's investigational gene therapy RP-L102 has earned both Fast Track and Regenerative Medicine…
According to a story from Reuters, the drug developer BioMarin is looking to make a name for itself in the hemophilia community in a very unique way. The company has…
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