According to a story from The New Paper, the KK Women's and Children's Hospital (KKH) in Singapore has successfully developed a test that could make diagnosing genetic rare diseases more…
Bone Therapeutics, who are developing an allogeneic bone cell therapy as a treatment for delayed-union fractures of long bones, has announced the results of a Phase 1/11A study. They have…
Three large black and white beasts rest under a shade tree to avoid the sun’s hot rays. They appear to be chewing gum, although they are really chewing their cud.…
According to a story from Hematology Times, a recent study found that genetic sequencing after the completion of a hematopoietic stem cell transplant could provide doctors with vital information about…
According to a report by CheckOrphan, a recent study investigated the use of genome editing directly in the body. The research involved the use of these editing techniques in cases…
The United States Food and Drug Administration has awarded Fast Track Designation to the investigational drug miransertib (ARQ 092), which is being researched as a potential treatment for PIK3CA-related overgrowth…
The process of getting diagnosed with a rare disease, as well as living with one, can be a traumatic experience for patients. Many rare disorders or conditions may commonly cause…
According to a story from pm360online.com, the biopharmaceutical company Insmed Incorporated recently announced that the data from its Phase 3 clinical trial of amikacin liposome inhalation suspension (ALIS). ALIS was…
Recent medical developments have led to new drugs designed to treat rare genetic disorders. However, the high cost of many of these drugs limits the number of patients who have…
Azathioprine is a drug used in the treatment of a variety of conditions. Inflammatory bowel disease, arthritis, and vasculitis may all include azathioprine as part of their respective therapies. It…
The National Organisation for Rare Disorders has announced the addition of ten new rare disease registries. For more detailed information about this news, you can view NORD’s source press release…
The Amyloidosis Foundation is a non-profit organization that is committed to working and advocating for the amyloidosis patient community. The foundation is dedicated to supporting amyloidosis patients and their families…
According to a story from BioPortfolio, the immunotherapy drug Kymriah, a type of CAR-T cell therapy, will be funded by NHS England for the treatment of patients with acute lymphoblastic…
Happy Friday! While the weather this week has been tumultuous, treatment news offers hope to the rare community. This week, we have an announcement about a Scleroderma registry, and PKU…
According to a story from Medical Xpress, a team of researchers has discovered a naturally occurring plant compound that exploits a particular weakness found in uveal melanoma, a rare type…
I’m writing this article now because it was something that I needed one year ago and didn’t have. I didn’t know anyone who had moved abroad with Common Variable Immunodeficiency…
According to a story from BioSpace, the biopharmaceutical company Soleno Therapeutics recently announced that it will be updating the minimum age for participating patients in its Phase III clinical trial…
Allison McElvin had a healthy pregnancy and all of her ultrasounds went well, so no one suspected anything about her little baby Cooper. Even when she had little Cooper…
For any football fans here, Matt Millen needs no introduction. For the rest of us who are unfamiliar, Matt is a four-time Super Bowl-winning linebacker who has played for the Oakland…
According to a story from techcentral.ie, the company OpenApp, which is based in Dublin, Ireland, has a developed a new online system that will allow for "virtual panels" of disease…
According to a report by the New York Times, a group of researchers tested the health benefits of bacteria this summer. Specifically, researchers aimed to determine if they were able…
According to a story from The Guardian, the viral disease monkeypox has been reported in the UK for the first time. The first patient was diagnosed on September 8th and…
According to a story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…
September is Primary Biliary Cholangitis Awareness Month! What is PBC? Primary biliary cholangitis (PBC) is a chronic disease of the liver characterized by a slow, progressive damage to the bile ducts…
According to a story from the Irish Examiner, treatment with the recently approved drug patisiran helped a British surgeon named Carlos Heras-Palou save both his hands and his career. Patisiran…
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