A recent interview with David Alvarez and his mother in the CTPost explains how he manages to remain positive after being diagnosed with spinal muscular atrophy (SMA). David is a freshman…
According to a story from BioPortfolio, Takeda Pharmaceutical Company Limited recently announced that its drug ADCETRIS (brentuximab vedotin) has seen its marketing authorization extended in the European Union. The European…
In a recent article appearing in BioPortfolio, Orphan Technologies announced a Phase 1/2 clinical trial involving the first Classical Homocystinuria (HCU) patients to be treated with OT-58. Twelve patients were enrolled as…
Loukisha shares her journey with hereditary angioedma (HAE). Living with a rare disease you'll experience highs and lows. Unfortunately the moments of struggle are unavoidable; all you can do is…
According to a story from Andre Eger, the drug developer Gilead recently had the misfortune to announce that its clinical trial testing an experimental drug for late stage nonalcoholic steatohepatitis…
According to a study published in the Orphanet Journal of Rare Diseases, a study that was intended to identify a new approach to monitoring the progression of x-linked adrenoleukodystrophy in women…
Content Warning: The following article discusses sexual abuse. According to a story from Buzzfeed News, many former patients of an esteemed medical researcher named Reginald Archibald, who was affiliated with…
In order to begin clinical trials for an experimental new drug, pharmaceutical companies in the U.S. are required to submit an Investigational New Drug (IND) application to the FDA. They…
According to a story from IT Business Net, the medical company KIYATEC, Inc., has recently announced that that the Knight Cancer Institute at Oregon Health & Science University has joined…
Tuberous Sclerosis Tuberous sclerosis complex (TSC) is a rare condition which results in the buildup of tumors in the skin, kidneys, brain, as well as other organs. Although the tumors are…
Inflammatory Bowel Disease Inflammatory bowel disease (IBD) is an autoimmune condition which causes inflammation throughout the digestive tract. There are two main forms of the condition- Crohn's disease and the…
Myalgic Encephalomyelitis/Chronic Fatigue Syndrome Myalgic Encephalomyelitis (ME) and Chronic Fatigue Syndrome (CFS) are interchangeable titles used to describe the same illness. Sometimes this illness is simply referred to as ME/CFS.…
Happy Valentine's Day! Whether you're looking forward to spending tomorrow with a partner, or with friends and family, we hope you're having a good week. Today, we're highlighting an about…
Gene Editing What is gene editing? It's been a hot topic over the last few years, but basically the concept is this: instead of disabling a malfunctioning gene, researchers are…
According to a story from Pharmacy Times, the US Food and Drug Administration (FDA) have recently approved the first ever therapy for the rare disorder acquired thrombotic thrombocytopenic purpura (aTTP),…
According to a story from pm360online.com, the Novartis company Advanced Accelerator Applications S.A. recently announced that Health Canada has issued approval for Lutathera™ as a treatment for gasteroenteropancreatic neuroendocrine tumors…
The Importance of Speech The ability to speak gives individuals not only the opportunity to communicate with one another, it provides an outlet for expression, creativity, and connectedness. Unfortunately, that…
According to a story from abc13.com, Washington state appears to be poised to become the third US state to ban dwarf tossing, a humiliating public spectacle used as entertainment in…
According to a story from abc13.com, Makenna Brown, a four-year-old girl from Texas, has been plagued by relentless itching thanks to her Alagille syndrome, a rare genetic disorder. Her distressed…
Duchenne Muscular Dystrophy Duchenne muscular dystrophy (DMD) is a rare, progressive, and fatal condition. It primarily affects males (1 out of every 3,600 to 6,000 male births). It's caused by…
Merkel Cell Carcinoma Merkel cell carcinoma is a rare and aggressive form of skin cancer. It's most often caused by the Merkel cell polyomavirus. However, it can also occur as…
According to a story from PR Newswire, the biopharmaceutical company Soligenix, Inc. recently announced that it has received a patent in the EU for the company's innate defense regulator drug…
Lambert-Eaton Myasthenic Syndrome Lambert-Eaton Myasthenic syndrome (LEMs) is a rare autoimmune disease. The body's immune system targets nerve cells meaning patients experience muscle weakness. Other symptoms of LEMs include difficulty walking…
According to a story from northjersey.com, Adam Levitz's life was hanging in the balance just a few weeks ago. His liver, which had endured years of damage from the rare…
According to a story from pm360online.com, The National Brain Tumor Society (NBTS) recently awarded $750,000 to the Global Coalition for Adaptive Research. This cash is slated to go towards the…
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