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…
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Ron Davis and his gene-sequencing technologies were mentioned years ago in The Atlantic alongside Elon Musk (SpaceX) and Jeff Bezos of Amazon fame. Since then, Davis has amassed over…
According to a story from westernmassnews.com, the community of Monson, Massachusetts is coming together to support Jason Harris and his family. Jason is ten years old and lives with Duchenne…
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…
According to a publication from Medical Laboratory Observer, a recently published case study following a family with Diamond-Blackfan anemia presents an overview of the condition; and a multi-step approach describing…
According to a story from BioPortfolio, the specialty pharmaceutical company Santhera Pharmaceuticals recently announced that it has submitted its marketing application to the European Medicines Agency (EMA) for its drug…
According to a publication on EurekAlert, the National Institutes of Health recently awarded Dr. Jason Karnes, PharmD, PhD (and assistant professor in the University of Arizona College of Pharmacy) with…
According to a story from brightsurf.com, familial hypercholesterolemia, a genetic condition that causes elevated levels of LDL cholesterol to appear at a young age, often goes undiagnosed. In fact, some…
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…
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Calls to Ban CRISPR A scientist named He Jiankui recently shocked the scientific world with his announcement that he had created twin “CRISPR babies”. The babies, a result of…
According to a story from blogs.biomedcentral.com, the findings from the IPF-PRO Registry, which is a US-based registry for idiopathic pulmonary fibrosis patients, were recently published. Patient registries are a critical…
According to a publication from Oncology Nurse Advisor, a study of 109 patients with myeloproliferative neoplasms (certain types of blood cancers) found that mortality rates for younger patients were similar…
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…
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