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…
According to a story from UPI, gene editing technology could be a potential treatment for mitochondrial diseases, a rare, genetic illness that is usually fatal. In a proof-of-concept experiment, researchers…
Show of hands - who has ever indulged in a little beauty pampering? A mani/pedi? A haircut? A shave? Sure, that was a trick question - who hasn’t?! And that’s…
According to a story from Angioedema News, an investigational product from BioCryst Pharmaceuticals called BCX7353 was able to quickly subdue swelling attacks in hereditary angioedema in clinical trials. In addition,…
According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration…
Attitude as defined in the dictionary: “A settled way of thinking or feeling about someone or something, typically one that is reflected in a person’s behavior.” In my opinion, how…
A study carried out by a research team at Yale University and mainly funded by the National Institutes of Health has identified a cell in the spinal fluid of patients…
According to a report by medicalexpress.com, researchers recently discovered a new genetic condition. Though researchers previously described the condition in animals, no human evidence had yet been found. Michigan State…
Michael Dickerson, aged 42, is originally from Hoedspruit Limpopo, South Africa. Recently, Michael was participating in a series of long distance trail runs when he learned about the story of Daniël…
According to a story from BioSpace, the U.S. Food and Drug Administration (FDA) recently rejected the approval of the drug Waylivra, which was in development for the treatment of familial…
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