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The FDA’s “untitled letter” to R3 Stem Cell (R3), based in Scottsdale Arizona, cautioned R3 that the product they are marketing is considered a drug. It must, therefore, be…
According to a story from the Madison Area Lyme Support Group, one of the most difficult challenges about Lyme disease is getting a confirmed diagnosis. Testing for Lyme disease is…
According to a story from kwqc.com, pediatric nurse Allee Mullen was devastated when her developing child was diagnosed with spina bifida, a rare and potentially debilitating birth defect. In order…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Anavex Life Sciences Corp. has just announced that the first patient has been dosed in its phase 2 clinical trial, which…
According to a story from abc.net.au, the local health services from northwest Queensland, Australia have recently been tasked with managing dialysis treatment centers in the region. This has improved access…
According to a story from Market Screener, the biotechnology company BioMarin Pharmaceutical Inc. recently announced that the Chinese government has approved the company's drug elosulfase alfa (marketed at Vimizim) as…
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…
According to a publication from Biospace, British clinical drug developer Orchard Therapeutics has secured global intellectual property rights to research, manufacture, and market a gene therapy program for the treatment…
According to a story from Market Screener, the drug developer RegeneRx Biopharmaceuticals, Inc. has recently announced that its licensee for RGN-137, an experimental wound healing gel, has begun the dosing…
According to a story from BioSpace, the drug developer Daiichi Sankyo Company Limited has recently announced positive data resulting from a pooled anaylsis of a phase 3 trial and a…
According to a publication from Benzinga, the U.S. Food and Drug Administration has granted Breakthrough Therapy status to Bayer's relapsed follicular lymphoma drug Aliqopa (copanlisib) for the treatment of marginal…
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 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…
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 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…
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…
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…
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.…
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