Claudia Stäubert and her team of researchers at the University of Leipzig have just reported noteworthy findings which could impact the future treatment of inflammatory diseases. Lactic acid bacteria, or…
Cluster Seizures Approximately 1/3 of all epilepsy patients live with uncontrolled seizures. Of these, around 150,000 in the United States alone experience cluster seizures (otherwise known as acute-repetitive seizures, crescendo…
A New AS Study A recent study, published in Reumatologia aimed to evaluate how ankylosing spondylitis (AS) patients are coping with their condition on a psychological level. They wanted to investigate…
Turner Syndrome Turner Syndrome (TS) is a rare disease caused by the partial or complete absence of the second sex chromosome. It is grossly underreported because 95% of fetuses who…
According to a story from the National Gaucher Foundation, the stress and difficulties of living with a rare or chronic disease can take a major toll on a patient's mental…
Huntington's Disease Huntington's disease (HD) is a rare neurodegenerative disease. It progressively affects the patient's mobility and cognitive function. Unfortunately, a cure has yet to be discovered and the condition…
According to a story from Financial Buzz, the drug developer Rafael Pharmaceuticals, Inc., recently announced that a Phase II clinical trial that will test the company's experimental drug CPI-613 in…
Happy Thursday! We hope everyone's enjoying the spring weather. Today, we're highlighting a story from a young man advocating for Huntington's patients. Next, we have an article about a surprising…
When you or your child is first diagnosed with a rare disease it can be so difficult to know where to turn. You want to learn everything about the condition,…
Orencia Orencia (abatacept) is a treatment for juvenile idiopathic arthritis (JIA) that was approved for use in the U.S. for the pediatric patient population in 2008. In Japan, the therapy…
According to a story from MD Magazine, a recent study that was presented at Digestive Disease Week 2019 suggests that patients with ulcerative colitis, a rare form of inflammatory bowel…
According to a story from The Washington Post, a host of different factors ultimately led to the tragic death of Olivia Shea Paregol, a freshman at the University of Maryland.…
According to a publication from Express Digest, Britain's National Health Service (NHS) has finally reached an agreement with American biotechnology company Biogen Inc. over the price of Spinraza - a…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
Short Bowel Syndrome Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
Coronado is a resort city located in San Diego, California. It's known for its luxury hotels, delicious restaurants, beautiful parks, and gorgeous beaches. It has just been announced that the…
SCID Severe combined immunodeficiency (SCID) is a rare disease which causes patients to have little to no immune response. With practically no immune system, SCID patients are very susceptible to…
The Origins of Fighting H.A.R.D. The Fighting H.A.R.D. Foundation was established in 2015 by two sisters who both live with multiple rare diagnoses. 16-year-old Allie is diagnosed with juvenile rheumatoid…
PREFER PREFER stands for Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle. It is a multi-stage project developed by Newcastle University's Welcome Centre for Mitochondrial Research in effort…
Phage Therapy Phages, or bacteriophages, are the natural enemy of viruses. They are the most abundant organisms in the biosphere. Researchers have used phages as treatments before, however after the…
According to a publication from the State University of New York at Buffalo, most symptoms of proximal renal tubular acidosis do not stem from the acidic blood levels that characterize…
According to a story from BioSpace, a Phase 1/2 clinical trial testing SGT-001, an investigational treatment for Duchenne muscular dystrophy, has been plagued by problems with side effects and adverse…
The Growth of Precision Medicine Precision medicine is the idea that each patient is an individual, and therefore deserves an individualized treatment plan to secure the best outcomes. It is…
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