September is Achalasia Awareness Month! Achalasia may not be a disease you've heard about - so you've come to the right place! What is achalasia? Achalasia is a rare disorder that…
September is Krabbe Disease Awareness Month! Because a lot of times, these rare diseases are rarely talked about, it'll be useful to deep dive into the disease to learn more…
The United States Food and Drug Administration has awarded Fast Track Designation to an experimental gene therapy for the treatment of wet age-related macular degeneration. For more detailed information, you…
According to a story from pm360online.com, the global biotechnology company Shire plc recently announced its product Takhzyro (injectable lanadelumab) was approved by Health Canada after priority review. Takhzyro is indicated…
Happy Friday! As the week comes to a close, we want to spotlight four articles. We have advice from a patient with CVID who learned how to manage her condition…
According to a story from The Health Site, National Eye Donation Fortnight began in India on August 25th and will continue until September 8th. There are over a million people…
According to a story from The New York Times, it didn't take long after Bertrand Might was born for his parents to realize that something was off. He had developmental…
A bill (H.R. 6801) has been introduced that is designed to support the development of rare disease therapeutics. Known as the Rare Disease Fund Act, it was introduced by Congressmen…
According to a story from news-medical.net, the new Evergreen Life health and medicines app could be a major benefit for patients across the UK, particularly patients with rare, chronic diseases…
According to a story from blogs.bmj.com, the situation has been tense recently in the halls of the Cochrane Collaboration. Cochrane is a non-profit organization that involves the work of over…
According to a story from pm360online.com, the drug development company Cytori Therapeutics recently announced that its investigational product ATI-1123 has been granted Orphan Drug designation from the US Food and…
According to a story from BBC, there are only around ten people in the world who are currently living with a replacement bladder that was derived from their own cells.…
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…
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