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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…
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