According to a story from PBS, the FDA has been approving new treatments at a noticeably faster rate than it has in the past. As a result, it appears that…
Happy Friday, everyone! We hope everyone's drinking water, wearing sunscreen, and taking a little time to relax this summer. As we get ready for the weekend, we've gathered four recent…
According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…
According to a story from NPR, plastic straw bans have been gaining traction across the U.S. One of the most shocking examples of planetary degradation as the result of human…
According to a story from Global News, direct-to-consumer genetic tests are becoming increasingly popular. These tests can be used to learn new info about your ancient ancestors, closer relatives, and,…
According to a story from pm360online.com, a new study of Prader-Willi syndrome will soon be under way thanks to a recent collaboration between the biopharmaceutical company Zafgen and the Foundation…
Duncan Avery is father to two children, Kalea, and Noah. At age six, doctors discovered Kalea had a tumor in her brain. They would successfully remove it via surgical methods.…
According to a story from medicalxpress.com, early tests of a gene therapy for X-linked myotubular myopathy are showing promising results. This could represent a major breakthrough for this disease, which…
Happy Independence Day Weekend! As the firework shows wind down, we want to wrap up the week with three great contributor stories, and one research update. First, we have some…
In 2001, when I was three years old, my dad was diagnosed with Adrenoleukodystrophy (ALD). ALD is a rare genetic neurological disorder with no effective treatment or cure. Over the…
According to a story from inpublic.globenewswire.com, the U.S. Food and Drug Administration has recently transferred its Orphan Drug designation for dovitinib from to Oncology Venture (OV) from Novartis, according to…
According to Queenstown Research Week, AWCBR, and QMB will be hosting a satellite event in August. The event will center on rare brain diseases, bringing together a group of dedicated…
According to a story from Check Orphan, a group of scientists from Stanford have begun the process of developing a new test that could help patients with rare Fanconi anemia…
According to a story from reuters.com, the U.S.-Israeli company BrainStorm Therapeutics will not provide its investigational stem cell treatment under the recently passed U.S. Right to Try law. The company…
The Australian government will give $33 million to researchers to study rare cancers and rare diseases. The original article can be read here, at the Department of Health’s website. Background…
Happy end-of-June, readers! A lot of people are getting ready for the fourth of July right now. However, as a lot of the rare community knows, not everyone is able…
According to a story from news-medical.net, a group of scientists from the Cincinnati Children's Cancer and Blood Diseases Institute have recently published a study in the journal Nature Cell Biology. This…
Jim and Melea Martin are Outreach Partners with Future by Design, a family support group organized by the Cystinosis Research Network. Melea has cystinosis and Jim is her husband. We…
This article discusses the plot of the biographical film Brain on Fire. Susannah Cahalan was twenty-four when she began experiencing seizures, hallucinations, and, after only a few weeks, catatonia. Her…
Tomorrow is World Scleroderma Day! This date is an opportunity for anybody with an interest in scleroderma to join together as one united voice to shine a spotlight on a…
According to a story from today.com, Dr. David Fajgenbaum was first diagnosed with Castleman disease in 2010. His first encounter with the illness was terrifying. He was confined to the…
Visit any pediatric ward in a hospital or medical center and you will see many pieces of medical equipment, but visit the OT and PT departments and you are in…
According to Sleep Review, researchers at the University of North Carolina School of Medicine are studying DNA repair after damage from anti-cancer drugs as well as DNA repair and how…
Although having a child with disabilities is no doubt stressful, the stress and worry does not necessarily lessen as the child grows older. As children with disabilities age towards adulthood…
According to a story from Fierce Pharma, the National Institute for Health Care and Excellence (NICE) has decided to reject the drug Crysvita for coverage on the National Health Service…
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