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…
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…
Amyotrophic lateral sclerosis (ALS) is a condition which affects the bodies nerve cells. It's neurodegenerative and progressive, causing the patient muscle weakness and muscle atrophy. It eventually results in paralysis.…
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…
President Herbert Walker Bush passed away on Friday, November 30th at the age of 94. News coverage of his life has reminded us all of the great legacy he leaves…
According to a story from Stanford News, a team of researchers affiliated with Stanford University have successfully developed a new technique for studying high-grade serous ovarian cancer, which is one…
According to a story from BioPortfolio, the drug developer Novartis recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational gene therapy AVXS-101, also known…
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…
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