According to a story from the CushieBlog, a recent study has found that a certain surgical procedure may be the best course of treatment for patients with Cushing's disease whose…
According to a story from Healio, Dr. Soumya Chatterjee of the Cleveland Clinic is convinced that the best way forward for treating systemic sclerosis (also known as scleroderma) is autologous…
According to a story from the website of Oregon rep Earl Blumenauer, Blumenauer (D-OR) along with fellow congressmen George Holding (R-NC) and G. K. Butterfield (D-NC) have introduced a bill…
The Beginnings Anne Pariser worked for 16 years at the FDA. For part of that time, she was a team leader for the Center for Drug Evaluation and Research where…
According to a story from BioSpace, the biopharmaceutical company Imara, Inc. has recently announced that its experimental product candidate IMR-687 has earned Fast Track designation from the US Food and…
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It has been nine years since Pfizer acquired the drugs vyndagel and vyndamax. According to a report in Biospace, based on the FDA’s recent approval of the two drugs to treat transthyretin-mediated…
According to a story from BioSpace, the drug developer Cadent Therapeutics recently announced that its investigational product candidate CAD-1883 has earned Orphan Drug Designation from the US Food and Drug…
According to a story from MD Magazine, the field of scleroderma treatment has seen some recent developments that could have major implications for how patients manage their disease. The latest…
For those who haven't finished watching HBO's Game of Thrones, this article contains spoilers for the series finale! If you don't want the ending ruined, read no further! Well, that…
Lynsey Chediak recently penned a story in Qrius about the future of rare disease treatment. Lynsey was born with arthrogryposis, a rare congenital condition in which scarring of muscle tissue…
It has just been announced that the very first national registry for chronic kidney disease (CKD) patients (at all stages of disease) will be launched next year. The creation of…
Happy Thursday! Today, we're highlighting two patient stories on adjusting to life and staying strong after a rare diagnosis. Next, we have an article about parents of children with Batten…
According to a story from CBS Philly, Jackson Guernsey, who is just two years old, was diagnosed with rare leukodystrophy just six months ago. While the disease has been taking…
The FDA has just approved the very first therapy for malignant pleural mesothelioma (MPM) in more than 15 years. It is the NovoTTF-100L System, developed by Novocure. Not only is…
According to a story from cbc.ca, Adrienne Mahoney was alive for over a year in a half without a pulse. The only thing that kept her alive was a special…
According to a story from Express Digest, Lucy and Mike Carroll don't know for how much longer their children will be able to live happy lives. This is because their…
A story from apsense.com provides a brief overview of signs, symptoms, diagnosis, and treatment approaches for Addison's disease, a condition also known as primary adrenal insufficiency. It is characterized by…
According to a story from ANCA Vasculitis News, the drug developer InflaRx has recently announced that the first patient has been dosed with IFX-1, an experimental therapy which is being…
Gunnar Esiason, a 28-year-old cystic fibrosis (CF) patient, just delivered an inspirational pre-commencement speech to the 2019 St. Louis University School of Medicine graduating class. Quite frankly, it contains wisdom…
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…
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