According to a story from PR Newswire, the Novartis Company AveXis has issued an announcement detailing special access programs that will help patients access Zolgensma, the first ever single use…
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CRISPR-Cas9, the genome editing tool, has caused quite a stir in the scientific community. According to a recent article in Science Daily, with results published in Nature Communications, CRISPR has…
Preclinical data supporting the efficacy of GKT831 as a potential therapy for cholestatic fibrosis has just been published in the Journal of Hepatology. GKT831 GKT831 is both a NOX1 enzyme and…
According to a story from globenewswire.com, the biopharmaceutical company Cellectar Biosciences, Inc. has announced plans to expand the third treatment cohort in its ongoing phase 2 clinical trial. This clinical…
According to a publication from Laboratory Equipment, a new animal study of mice with simulated Huntington's disease suggests that a protein associated with the symptoms of Huntington's can form small,…
According to a story from PR Newswire, the Novartis company AveXis recently announced that the US Food and Drug Administration (FDA) has approved Zolgensma, making it the first ever gene…
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Newly diagnosed patients may decide to either receive medical treatment or forego treatment based on the results of their diagnostic test. As recently reported in PEW, although it is…
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…
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