According to a story from globenewswire.com, the gene and cell therapy company Abeona Therapeutics Inc. recently announced that has received a letter from the US Food and Drug Administration (FDA)…
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The accelerated approval granted by the FDA for the Duchenne muscular dystrophy drug eteplirsen, developed by Sarepta Therapeutics, was granted only after internal disagreements and protests had been resolved.…
A minimum of seven people have died after contracting Eastern Equine Encephalitis (EEE), a rare disease carried by mosquitoes, bringing number of total people affected to 27 in six states.…
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Fierce Biotech recently focused on a report by The BMJ, a British science journal, on the validity of clinical trials which are critical to decision making by patients and…
Tay-Sachs Disease September is Tay-Sachs Disease Awareness Month. Voted on by the Senate unanimously in 2008, this month aims to spread awareness about this disease and the importance of carrier…
According to a story from scienmag.com, a pair of five year grants that were awarded to Dr. Mark Zylka will go towards projects focused on autism and the rare disease…
According to a story from the Washington Post, Bob Schwartz is a walking, talking medical mystery. He lives with a strange and diverse array of difficult symptoms, and after being…
According to a story from Benzinga, the medical device company V-Wave Ltd. has recently announced that a product in development by the company has earned Breakthrough Device designation from the…
According to a press release from the California-based biotechnology company Ardelyx, the US Food and Drug Administration (FDA) recently approved the company's irritable bowel syndrome drug candidate Ibsrela (generic name…
According to a press release from CTD Holdings, the American Food and Drug Administration (FDA) recently approved an Expanded Access application from a physician to treat a pediatric Niemann-Pick disease…
According to a press release from global pharmaceuticals titan GlaxoSmithKline, the US Food and Drug Administration (FDA) recently expanded approval of the Company's eosinophilic asthma treatment Nucala (generic name mepolizumab)…
There are currently 11 treatment options for multiple sclerosis (MS) in the United States. Some of these are symptomatic treatments, and some actually slow progression of the disease. Before 2009…
A recent study, published in Nature, examined whether or not stem cells could be modified to reduce their chance of rejection. Stem cell therapies are utilized in a wide array…
According to a recent publication from Express Digest, England's National Health Service (NHS) has reversed its earlier position on a rare disease drug after lengthy public debate, now agreeing to…
Happy Thursday everyone! We're seeing more and more leaves falling over here, and the weather is genuinely beinning to cool down. Today, we're lighting a two part patient story covering…
Bob Hussey Bob Hussey is a 62-year-old mechanical engineer who, thanks to his own experience as an ulcerative colitis patient, has created a new invention that could help a wide…
According to a story from ir.stockpr.com, the biotechnology company CTD Holdings, Inc., recently announced that the US Food and Drug Administration (FDA) has issued an approval for an individual Investigational…
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The city of Jacobina, Brazil in the Bahia region was selected as the ideal area for a study designed to control disease-bearing mosquitoes. A recent report in the Science…
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Diffuse large B-cell lymphoma (DLBCL) is a common type of non-Hodgkins lymphoma. World Pharma News recently reported that the FDA approved the first chemoimmunotherapy, Polivy, for DLBCL in combination with…
Patient Worthy has written extensively about Dr. David Fajgenbaum, who has made it his life's mission to find a cure for Castleman Disease. Dr. Fajgenbaum is not only diagnosed with…
According to an announcement from the International Waldenstrom's Macroglobulinemia Foundation (IWMF), the organization has recently added a new fact sheet to their website. This fact sheet is focused on the…
According to a story from BioSpace, Janssen Pharmaceutical Companies recently announced that the European Commission (EC) has expanded the approved indication of the company's drug ustekinumab (marketed as Stelara). This…
According to a story from newswise.com, the American Neuromuscular Foundation has recently awarded a grant to Dr. Karissa Gable. This grant will go towards a research project headed by Dr.…
According to a story from BioSpace, the genetic medicines company Passage Bio recently announced plans for its sixth program for gene therapy development. The company plans to work with the…
We're almost halfway through September believe it or not! As autumn approaches, we're continuing the aggregate rare disease news. This week, we're highlighting three studies, and one article on a…
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