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…
Behold the power of sheer will, determination, and tenacity! New York teen Hannea Milliman overcame the rare disorder Parsonage Turner Syndrome - which causes severe pain in the shoulder and arm -…
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…
According to a story from statnews.com, the recent FDA approval of a new treatment for Lambert-Eaton myasthenic syndrome could actually be bad news for patients. As it turns out, the…
As Leah approached her late twenties she was, in her own words, a “tank.” She kept up with her ex-Marine husband’s work outs and ran every day. She was the…
As part of the NFL's "My Cause, My Cleats" awareness project, New York Giants Running back Saquon Barkley chose 22q11.2 deletion syndrome (22q) as his cause to highlight! And while that…
The first week of December is Crohn's & Colitis Awareness Week! Courtesy of the Crohn's & Colitis Foundation, there are a few things we can do as rare disease advocates to…
According to a story from Grimsby Live, the family of six year old Melissa Stark, who has Williams syndrome, has transformed their house into a Santa Claus themed wonderland of…
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