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…
According to a story from Medical Xpress, a new monitoring device called the STAT-ON will allow physicians to make more informed decisions regarding changes to treatment for Parkinson's disease patients.…
Living with a chronic long-term health condition, can have a devastating effect on someone’s life as it affects everything we do. It’s like finding yourself on a new path without…
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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…
I remember when I was younger and I felt so carefree. My life was easy-breezy, and full of great times, cool friends, laughter, and a go-with-the-flow attitude. I really didn’t…
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,…
A publication at AP Sense outlines some important steps of the early identification and treatment of Addison's disease, a rare condition brought about by the body's underproduction of certain hormones.…
According to a story from Boehringer Ingelheim Pharmaceuticals, Inc., the company has recently announced positive results from a phase 3 clinical trial testing the company's drug nintedanib (marketed as Ofev)…
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
According to a story from Science Daily, a review of studies focused on allogeneic stem cell transplants as a treatment for non-Hodgkin lymphoma found that it wasn't possible to conclude…
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…
According to a publication from Science Alert, a recent study headed by Dr. Simon Jiang suggests that two rare genetic mutations may be responsible for most cases of lupus. The…
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…
According to a story from EurekAlert!, a team of researchers studying lupus nephritis, a kidney disease that can affect people diagnosed with lupus, are utilizing a highly precise new tool…
According to a story from BioPortfolio, the biopharmaceutical company Clovis Oncology, Inc. recently released data from a phase 3 clinical trial of patients with recurrent ovarian cancer taking the company's…
June is myasthenia gravis awareness month! Myasthenia gravis (MG) is characterized by weakness and rapid fatigue of any of the muscles under voluntary control. It's caused by a breakdown in…
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 publication from People, Sara Geurts, a 28-year-old model with Ehlers-Danlos syndrome, has been bedridden for several weeks due to complications linked to her condition. Geurts, a Minneapolis…
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Although somewhat of an exaggeration, the title of this article falls in line with the thinking of Insitro’s founder, Daphne Koller. In a recent interview with Bloomberg, Koller said that…
According to a story from apnews.com, the gene and cell therapy company Abeona Therapeutics, Inc., has recently announced that the US Food and Drug Administration (FDA) has given the company's…
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