Happy Thursday! Today, we're highlighting a piece from a PW contributor sharing why it's important for people in the rare disease community to vent sometimes. If you'd like to use…
According to a story from BioPortfolio, AstraZeneca and Merck have recently announced the results from a phase 3 clinical trial testing olaparib (marketed as LYNPARZA®) as a treatment for BRCA…
According to a publication from Scleroderma News, a study recently published in Clinical & Transitional Immunology suggests that certain cytokines (small proteins that play an important role in communication between…
According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from…
The Problem One in every 12 Canadians will be diagnosed with a rare disease in their lifetime. Two/thirds of these individuals are children and most of these patients experience debilitating…
Sydney Mccallister is a 32-year-old woman diagnosed with spina bifida. Essentially, the condition means that the neural tube has not closed all the way during the first month of embryonic…
Passage Bio has just recently announced that Krabbe disease will be the focus of their third clinical trial program. Krabbe Disease Krabbe disease is a rare, inherited, neurodegenerative lysosomal storage…
According to a story from statnews.com, one pharmaceutical company is taking advantage of a little known loophole in the Orphan Drug Act to suppress competition and rake in gluttonous profits.…
According to a story from Science Daily, a team of scientists from Johns Hopkins Medicine conducted a review of studies going back nearly 50 years and have concluded that surgery…
In a letter to Dr. Norman Sharpless, the acting commissioner of the Food and Drug Administration since April 5, Senator Ted Cruz encouraged the federal agency to expand the number…
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 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…
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…
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 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…
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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…
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