Rare diseases come in many forms, shapes, and sizes - with varying degrees of symptoms and attributes. Here is one that made news on the Internet, for it's peculiar and…
According to a story from HD Buzz, a recent study has conducted an in-depth investigation into juvenile onset Huntington's disease. It is quite unusual for this juvenile onset form to…
According to a story from The Atlantic, the continual development of gene editing technologies like CRISPR have the potential to cure a substantial portion of rare diseases that are linked…
A drug called Coversin is being investigated as a potential treatment for four orphan diseases: bullous pemphigoid (BP), atypical keratoconjunctivitis (AKC), thrombotic microangiopathies, and paroxysmal nocturnal hemoglobinuria (PNH). Recently, new…
Happy October, everyone! We hope everyone's enjoying this new ~fall weather~ and the endless supply of pumpkin spice flavored food products. This week, we're highlighting four FDA announcements we think…
According to a story from 9news.com.au, the first sign that Jemima Leydon had ovarian cancer was a stomachache. Her mother Megan assumed that it was just a stomach bug that…
The clinical research process is a thorough and time consuming method in which new drugs, therapies, and medical devices are rigorously tested before being made available to the public. Through…
According to a story from MedCity News, a recent study revealed a potential weakness of CAR-T cell immunotherapies that reveals the need for precision and caution in the extraction process.…
A past, current, and future problem – underrepresentation in clinical trials, particularly for rare diseases. Why does representation matter? Besides, you know, the basics of equality, drugs can react differently…
According to a story from wtvbam.com, the drug development company Alnylam Pharmaceuticals has recently stated that it is looking less likely that its gene silencing therapy givosiran will be getting…
Ultragenyx Pharmaceuticals has shared data from two cohorts taking part in an on-going study involving an investigational gene therapy for ornithine transcarbamylase deficiency. For more information, you can view the…
Topline results have been shared from a Phase 3 study of the investigational drug givosiran in patients with acute hepatic porphyria. For more detailed information you can view the source…
Many people express skepticism about the pharmaceutical industry. If not skepticism, than concern, if not wholesale distrust. For every few negative stories, however, there is at least one positive example.…
Many persons living with rare disease require a transplant of certain organs. Though this is not a cure for the rare disease Cystinosis, a kidney transplant is often required once the disease…
According to a story from Parkinson's News Today, a recent analysis of Parkinson's disease research showed that scientists would benefit from relying less on animal models and instead should focus…
That title. How can blessing and POTS even coexist in the same sentence? Well, my journey started in 2000 when I began noticing strange things happening in my body: intense…
Over the weekend, the New York Times ran an opinion piece that caught our attention. "He Got Schizophrenia. He Got Cancer. And Then He Got Cured." Click here to read…
Researchers at the University of Utah are working on developing computational tools that can be used to help identify the genetic changes underlying early infantile epileptic encephalopathy. For more detailed…
According to a story from news-medical.net, a genomics study called Angiosarcoma Project has successfully discovered that the inhibition of immune checkpoints could have therapeutic value for patients with angiosarcomas affecting…
According to a story from the Milton Keynes Citizen, a local boy named Josh from the Milton Keynes community with the rare lysosomal storage disease Niemann-Pick disease type C will…
Shriners Hospitals for Children® has announced their motion analysis centre consortium. The hospital’s Motion Analysis Centre is used to evaluate the movements and gait of children and teenagers. To read about…
Joe Marksteiner, a 22-year Air Force veteran and former gymnast, won a title at the 2013 World Powerlifting Championships at the age of 60. This year, now at 65, he…
According to a story from Medical Xpress, an international group of scientists from the US, France, and Spain may have discovered that an antifungal agent commonly used to treat skin…
The United States Food and Drug Administration has awarded Orphan Drug Designation to an experimental PLX cell therapy (called PLX-R18) as a treatment for graft failure and incomplete haematopoietic recovery…
Happy Friday, everyone! We're trying something new this week. Instead of wrapping up with an Editor's Choice article, we're highlighting four FDA announcements we think you should know. Check them…
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