According to a story from Charcot-Marie-Tooth News, the continuously escalating cost of drugs in the United States is still plaguing rare disease patients and chronically ill people across the country.…
Some of you may have heard this story, others may be completely new to this, but bear with us on this funny and on-topic story that made the rounds on…
When it comes to rare diseases often one of the biggest hurdles is finding the right diagnosis. Tailoring treatment, and adjusting lifestyles can be difficult, but both are impossible if…
The Scottish Medicines Consortium, a committee that advises NHS Scotland on what medicines they should provide, has accepted four new drugs for use. These are gemtuzumab ozogamicin for acute myeloid…
Getting access to medication can be a difficult and demanding process for patients. There are often a number of hoops to jump through, papers to sign, tests to go through…
According to a story from MD Magazine, researchers are using real-world studies, outside of the controls found in the clinical setting, to trace the impacts of multiple recently approved therapies…
According to a story from The Examiner, Sarah Nauser-Olsen was at a great place in her life: she had a stable job as a police officer and was also well…
Facebook has been making changes to how content will be shared on people’s newsfeeds. These are designed to help users connect more with friends and family, and limit the number…
According to a story from Xcomony, the tech startup Glympse Bio has been developing a sensor technology that it claims will allow doctors to detect a variety of diseases earlier…
Hannah Evans, a 27-year-old woman from South Wales with several rare diseases, has reached her £37,500 fundraising goal that will support her trip to America to access an experimental and…
October is Trigeminal Neuralgia Awareness Month! One way you can honor this month is by signing TNnME: Intl Trigeminal Neuralgia and Me's petition for the World Health Organization (WHO) to add Trigeminal Neuralgia…
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…
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