Much of what we think counts as "treatment" in medical literature is informed by the amazing successes of high-tech drugs and therapies that have evolved in just the past few…
According to a story from Ankylosing Spondylitis News, a recent study has reaffirmed the understanding that patients with ankylosing spondylitis, a rare form of inflammatory disease that affects the joints…
According to a story from Lambert-Eaton News, a recent large scale study using data from the Veterans Affairs has determined that the prevalence of Lambert-Eaton myasthenic syndrome (LEMS) is similar…
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A recent posting by the Leukemia and Lymphoma Society references the Beat AML Master Trial. The trial evaluates targeted therapies to treat acute myeloid leukemia (AML) and was launched…
Uveitis Uveitis is a rare condition in which the uvea of the eye becomes inflamed. The uvea is responsible for providing the majority of the blood to the retina and…
According to a story from People Magazine, parents Deborah and Antoine Vauclare were devastated when they found out that their son Léo was born with an incredibly rare genetic disorder called infantile…
According to a story from globenewswire.com, the biopharmaceutical company Innovation Pharmaceuticals recently released a report in which recent studies and pre-clinical research has suggested that the company's experimental drug Kevetrin…
An Unexpected Emergency Kristin Hardy and her sister Margaret are no strangers to rare disease. Kristin has primary progressive multiple sclerosis (MS) and Margaret has relapsing-remitting multiple sclerosis. Margaret also…
Happy Thursday! This week we're highlight a variety of articles. First, we're starting with a strong call-to-action from contributor and advocate, Sharon. Next, we have an article about rare disease…
We just read exciting news for the SCID community from Stanford researchers. The original study was published in Nature Communications, but a summary appeared in Medical Xpress. The researchers have…
According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular…
After 20 agonizing years, a mother and father have finally received answers regarding their daughter's health. It shouldn't take this long. The Diagnosis Their daughter was diagnosed with two very…
According to a story from Healio, a recent study has found that 58 percent of patients with the inflammatory disease ankylosing spondylitis will experience relapse within one year after stopping…
Sarcoidosis Sarcoidosis is a rare inflammatory condition that causes granulomas to buildup within the organs. These can cause permanent organ damage. While the condition can affect various organs, and presents…
We need to talk about adult rare disease patients. Adults who have a rare disease diagnosis are faced with a blaring issue-- I'm speaking for a large number of adults here,…
According to a story from finanzen.ch, the pharmaceutical company Eisai, Inc. announced that that company presented a total of four abstracts which were related to a Phase 3 clinical trial…
According to a story from the Andover Advertiser, mother June Beere has kicked off a campaign to open a cancer support center after her 42 year old daughter Gemma Kent…
According to a story from Yahoo, the family of Wyatt Beiermann, a seven year old boy with lethal mitochondrial disease, is doing their best to savor the limited time that…
According to a story from The Del Mar Times, the Susan Poorman Blackie Ovarian Cancer Foundation (SPB Foundation) and the Clearity Foundation have recently partnered in order to facilitate a…
According to a story from express.co.uk, Elle Morris was only 11 years old when she died from a severe lung infection. Elle was born with cystic fibrosis, a serious genetic…
According to a story from jsonline.com, Tyler Trent, a Purdue University student who captured the hearts of thousands with his struggle against osteosarcoma, passed away at age 20 after his…
According to a story from the Edinburgh Evening News, Stephen Ritchie of Edinburgh, Scotland, has been an active participant in a progressive multiple sclerosis clinical trial since November of last…
Doctor Alan Pestronk has identified a new and very rare autoimmune disease. He is now trying to spread the word about its existence to other doctors in an effort to…
Noonan Syndrome Noonan syndrome (NS) is a rare disorder that is typically diagnosed at birth. It affects the child's development resulting in stunted growth, abnormal facial features, and heart defects,…
According to a story from BioPortfolio, the biotechnology company Tiziana Life Sciences plc recently announced encouraging results from an interim safety review of the company's Phase 2a clinical trial. This…
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