A recent study published in Acta Neuropathologica discusses the importance of symptom and health management for people with progressive supranuclear palsy. The rare condition, which causes neuronal degeneration, has no cure. But…
It is no secret that COVID-19 has turned life as we know it upside down. It has changed everything from hanging out with friends to working to going grocery shopping.…
A study in the American Journal of Hematology recently presented some troubling findings on the relationship between Fanconi anemia and allogeneic hematopoietic stem cell transplantation (alloHSCT). Patients who participated in alloHSCT before achieving…
For those in the rare disease community, you know that it can take a long time to reach a diagnosis for your condition: an average of 4.8 years! But upon diagnosis, many…
Disposable Scopes Disposable scopes are exactly as they sound. Medical scopes utilized for the bladder, lungs, colon, etc. can be made reusable or disposable. Disposable scopes allow faster and more…
A recent news release on BioSpace shares how GTX-102, an investigational antisense oligonucleotide drug therapy for Angelman syndrome, was granted Fast Track designation from the FDA. The therapy, created by GeneTx…
Ana Pina from the Centro de Estudos de Doencas Cronicas at NOVA Medical School in Portugal, and her colleagues recently published an innovative study regarding the diagnosis of familial hypercholesterolemia (FH).…
The last decades have seen significant progress when it comes to medical care for rare diseases. Tasks doctors once slaved over were transformed with better speed and efficiency by modern…
Edwin Chapman, who works at the University of Wisconsin-Madison, has been studying neurons for most of his career. He and his team recently made a discovery regarding SYT1-associated neurodevelopmental disorder.…
Bridging the Voice of ALS Patients | ATSDR Bridging the Voice of ALS Patients Because learning more about ALS is an important step in the battle to defeat it, the…
Promising new drug target for autoimmune and allergic diseases revealed Credit: The Babraham Institute A study led by researchers at the Babraham Institute in collaboration with the Wellcome Sanger Institute…
Imagine that you're a parent of five beautiful daughters. One night, you put them all to bed and all seemed well: playful, happy, excitable. Then, the next morning, you wake…
Blood clots and fever and coughing: oh my! As COVID-19 spreads throughout the globe, researchers are discovering more and more symptoms associated with this novel coronavirus. But what happens after recovery? Well,…
In 2003, a respiratory illness called Severe Acute Respiratory Syndrome (SARS) swept the globe. The condition, caused by a coronavirus, spread throughout two dozen countries, infecting 8,098 people and killing…
Many patients with rare diseases require some sort of treatment or therapy to manage their conditions. In the case of atypical hemolytic-uremic syndrome, or aHUS, this therapy is dialysis. Learn…
By Natalie Homan from In The Cloud Copy The COVID-19 pandemic has changed the lives of millions around the world in an incredibly short span of time. It’s hard to…
By Natalie Homan from In The Cloud Copy Two businesses that advocate for patients with rare and orphan diseases announced recently that they are teaming up to help patients get…
According to Hemophilia News Today, data collected from a Phase 2b trial highlights that the subcutaneously administered DalcA is effective for the treatment of hemophilia B. Find the full press…
This was originally published as the Seventh Newsletter for the Leukodystrophy Resource Research Organisation (LRRO) on April 19th 2020. You can read more from their newsletter here. To state the blatantly…
Click here to read part 1 and here to read part 2 After coming back from the Global Genes conference, Kristie was convinced that establishing a foundation was the right…
May is recognized as Behçet's Disease Awareness Month and May 20th is recognized as Behçet's Disease Awareness Day. The focus of these events is to help spread awareness about this…
When studying biomarkers of lupus nephritis, researchers at the University of Houston discovered something interesting: race-based differences. Biomarkers are some sort of measurable substance; in this case, urinary proteins. However,…
Enanta Pharmaceuticals has recently released the results from the second phase of its INTREPID trial, which studied EDP-305 as a treatment for primary biliary cholangitis (PBC). The results were positive,…
According to CNN, the CDC recently sent out a health advisory to doctors across the United States alerting them of a new syndrome affecting children in eighteen states, Washington, D.C.,…
According to ANCA Vasculitis News, recent research shows that patients with multiple sclerosis who are being treated with Lemtrada are at greater risk of developing severe ANCA vasculitis. The drug therapy…
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