After years of having multiple physical complications and problems, a young woman in Spirit Lake found medical answers in 2016. In that year, Jessica Brown was diagnosed with Chiari…
According to a press release from Alnylam Pharmaceuticals, the Massachusetts-based biopharmaceutical company has completed the submission of its New Drug Application (NDA) for givosiran — its experimental acute hepatic porphyria…
According to a story from bioengineer.org, a team of researchers associated with Oregon State University are making further headway in understanding the connection between our health and the bacteria that…
Stem Cell Therapies While stem cell therapies hold a great deal of promise for many rare diseases, there is still a lot that we don't understand. Experimental stem cell therapies…
Meredith Resnick's breast cancer risk is 42%. This risk is made up of her higher breast density (denser breasts are associated with increased risk), Ashkenazi Jewish heritage, a family history…
"A new way to improve the efficacy of a drug taken by millions of patients throughout the world." Background Adalimumab (also known as Humira) and infliximab (also known as Remicade)…
According to a story from Bloomberg, the company Cadent Therapeutics recently announced that the company's experimental product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…
MVID Microvillus inclusion disease (MVID) is a rare, gastrointestinal disease which can cause impaired development in children. It is characterized by severe diarrhea which leads to dehydration and malnutrition. MVID…
Happy Thursday! Today, we're highlighting four articles on rare disease news. First, we have a story on a lawsuit against controversial stem cell treatments. Next, we have an article on…
Stargardt Disease Stargardt disease is a rare condition that causes vision loss due to the buildup of vitamin A byproducts in the retina. The byproduct causing the largest buildup is…
According to a publication from The Portland Press Herald, Judge Ursula Ungaro, a senior District Judge in the Southern District of Flordia, recently ruled in favor of the United States'…
According to a story from Acrofan, the biopharmaceutical company SpringWorks Therapeutics, Inc. has recently announced the that company's experimental product candidate PD-0325901 has earned Fast Track designation from the US…
A recent study published in PLOS Biology has shown a promising new vulnerability within human viruses which could lead to the development of a new antiviral drug. This study was…
Transfusion-Dependent β-Thalassemia (TDT) Transfusion-dependent β-thalassemia (TDT) is a rare disease that is caused by a mutated β-globin gene. This mutation causes the amount of hemoglobin in the body to be…
Did you know that people who have had cancer before are more likely to get a second cancer than people who have never had cancer at all? Cancer survivors…
With the advent of a majority conservative Supreme Court with the appointment of Brett Kavanaugh last year, several US states have recently passed laws that effectively ban abortions. Many of…
According to a story from BBC, 35 year old Kimberly Chard knows that she probably doesn't have much longer to live. Kimberly was born with cystic fibrosis, a rare disorder…
No one ever likes to think about worst possible scenarios. But sometimes by thinking about these things for a small period of time, we can make simple preparations which could…
The Danger of Stereotypes There is still a huge stereotype that women are prone to "hysteria." This leads to a general distrust in women's self-reported symptoms. Physicians consciously or unconsciously…
Systemic Sclerosis Systemic Sclerosis (Systemic Scleroderma) is a rare disease which results in the hardening of the connective tissues in the body as well as the skin. It can affect…
The PoppyPocket The PoppyPocket is a wearable pocket, created to make wearing infusion pumps and well as other medical devices easier, safer, and more comfortable. It comes with two pockets,…
According to a story from Business Wire, the drug developer Cadent Therapeutics recently announced that its investigational drug CAD-1883 has been awarded Orphan Drug Designation from the US Food and…
According to a story from kwqc.com, pediatric nurse Allee Mullen was devastated when her developing child was diagnosed with spina bifida, a rare and potentially debilitating birth defect. In order…
An Idea Sometimes life leads us down unexpected paths. Mark Greget founded a medical device distribution company. That was his plan. But after interacting with patients through his work, he…
The Beginning of the Journey Claudia Martinez is a medical student at UTHealth McGovern Medical School. Her dream is, and always has been, to be a doctor. But an unexpected…
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