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…
Do you have ADPKD? If you or someone you know has ADPKD, email us at [email protected]. We'd love to hear from you about your experience! According to a story from…
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In the mid-1980s the FDA was criticized for being slow in approving new drugs. In fact, a commission was formed as a result of the mounting criticism. Janet Woodcock,…
According to a story from ANCA Vasculitis News, a recent study conducted in Korea determined that ANCA vasculitis patients in the region were at a greater risk of developing thyroid…
"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…
According to a story from Science Nordic, the monoclonal antibody drug rituximab (often marketed at Rituxan) had no effect on patients with chronic fatigue syndrome (CFS). Chronic fatigue syndrome is…
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…
According to a story from abc.net.au, Todd Murfitt from Adelaide, Australia, was only 35 when he noticed that his left hand was starting to tremor as he typed. After a…
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 story from globenewswire.com, the genetic medicines company Homology Medicines, Inc. has recently announced that it has opened enrollment for its phase 1/2 clinical trial. This clinical trial…
According to a story from The ASCO Post, recent studies suggest that around 70,000 adolescents and young adults are diagnosed with some type of cancer in the US each year.…
According to a story from Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…
According to a story from mdmag.com, a recent study that was presented at the 2019 meeting of the American Society of Clinical Oncology has demonstrated the potential of a two…
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…
According to a story from Australasian Science Magazine, the dawn of the gene therapy "revolution" is upon us. While there are only a handful of gene therapies that have actually…
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…
About a month ago, we reported on a story about an Irish family that is facing deportation from Australia. The family includes Anthony and Christine Hyde along with their three…
According to a story from Scleroderma News, a team of researchers associated with Michigan Medicine has been awarded a grant to the tune of $10.2 million from the National Institutes…
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…
According to a press release from Danish biopharmaceutical company Orphazyme, the Company intents to proceed with a marketing authorisation application (MAA) for its experimental Niemann-Pick disease type C treatment arimoclomol.…
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