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.…
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 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…
According to a story from PR Newswire, presentations at the 60th annual American Society of Hematology Meeting and Exposition are highlighting the results of four large scale clinical trials that…
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…
My name is Julie Bell. I am someone living with CIDP, Believe or not, it’s changed my life. And today, I consider myself blessed—even despite having a rare and equally…
According to a story from einnews.com, the cell therapeutics company American Gene Technologies (AGT) has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation…
The Washington Post reports a gripping story of a family struck by a rare disease diagnosis, but also a heartening story of a global community coming together to support them…
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