Extended Collaboration Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…
Open Style Lab is an organization that considers itself dedicated to creating "functional wearable solutions for people of all abilities without compromising on style.” They use a wide variety…
We all run into problems in our everyday lives that cause frustrations and test our patience. If we're feeling well, we react to those issues at the moment and then…
According to a story from the South China Morning Post, there are an estimated 20 million people in China that are rare disease patients. The Chinese government has 121 diseases…
The Problem Orkambi is a drug for cystic fibrosis (CF) created by Vertex. It is effective for approximately 40% of all CF patients. It was licensed in 2015 by the…
According to a story from finanznachrichten.de, the biotherapeutics company 4D pharma has recently announced a research partnership with the MD Anderson Cancer Center at the University of Texas. The goal of…
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…
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