According to a study published in January in the scientific journal Acta Neurologica Scandinavica, the use of gamma knife surgery as a treatment for a rare tumor called a vestibular schwannoma can…
April is designated as Sarcoidosis Awareness Month. This year's event will be the sixth annual awareness campaign, which will involve collaboration between the Foundation for Sarcoidosis Research and the American…
Michael and Armené Kapamajian were at home in Los Angeles settling in with their one week old baby, Sasoun, when their doctor called and asked them to come in…
According to a study summarized in Gastroenterology Consultant, pregnant women with intrahepatic cholestasis of pregnancy are much more likely to also be diagnosed with nonalcoholic fatty liver disease. You can…
As initially covered by MyLondon News, the family of a London boy with spinal muscular atrophy (SMA) is looking to advocate for others with this genetic disorder. Additionally, they…
According to a release on Public News Service, the state of West Virginia just implemented a new bipartisan law to support individuals with rare diseases and their families, a particular…
According to a story from BioSpace, Acceleron Pharma Inc. and Bristol Myers Squibb have announced recently that the US Food and Drug Administration (FDA) has recently approved a new drug…
April 17th, 2020 is World Hemophilia Day. This event is held to help foster awareness about hemophilia and other related bleeding disorders. It is a day when this rare community…
The month of April is recognized as Parkinson's Disease Awareness Month. While Parkinson's disease is not considered rare, Patient Worthy chooses to continue to provide coverage of this illness because…
April 22nd, 2020 heralds the beginning of World Primary Immunodeficiency (PI) Week. This will be the ten year anniversary of the event, which is designed to spread awareness about PI…
April 15th, 2020 will be the 7th annual International Pompe Day, a day of recognition and fostering awareness around Pompe disease organized by the International Pompe Association. Like many rare…
CAR-T cell therapy has shown to be a viable treatment for mantle cell lymphoma after being studied at the University of Texas MD Anderson Cancer Center. The study showed that…
In this time of cancellations, closures, and shutdowns, many companies and individuals have found unique and creative ways to shift their services online. Better yet, some have temporarily waved their…
A recent study published in the Orphanet Journal of Rare Diseases examines the psychological burden of mitochondrial diseases in children that are suspected of having some form of it but have yet…
According to a story from Mirror Online, Vera Twomey from Cork, Ireland, was devastated when her four month old daughter had a severe seizure that lasted 45 minutes. Before long,…
According to a story from Hindawi, a recent study focused on congenital central hypoventilation syndrome serves to bring to light the importance of genetic testing of close family members based…
Como se informó originalmente en Global News., La adolescente de Nuevo Brunswick, Mackenzie Walsh, cumplirá 16 años esta semana, pero no quiere un automóvil o una dulce fiesta de 16.…
According to a story from EurekAlert!, a recent discovery related to myelin, a fatty acid that coats neurons, could have significant implications for diseases that impact it, such as multiple…
What is Repurposing? Repurposing involves testing whether or not a compound approved of another condition may be capable of also treating an illness with no approved treatment option. It's advantageous,…
According to a story from Vox, while the Center for Disease Control and Prevention (CDC) and other health authorities have reported the primary symptoms of coronavirus/COVID-19 to be a dry…
Dr. Pierre Fenaux, of Hôpital Saint Louis, Paris and lead author of the MEDALIST trial, told Ash Clinical News that treatment with the investigational drug, luspatercept, improved the rate of…
According to a story from The Columbus Dispatch, a variety of disorders, such as autism, can cause issues with sensory processing. What this means is that people on the autism…
According to a story from Sleep Review, the drug company Jazz Pharmaceuticals plc has recently filed a New Drug Application (NDA) for its investigational treatment JZP-258, which is being developed…
By now, everyone has heard of COVID-19. Information is constantly being updated surrounding the nature, symptoms, spread, and potential treatments for the illness. Despite this constant influx of information, there…
What is Homocystinuria? Classical homocystinuria, or HCU, is a rare metabolic condition which its caused by a deficiency of the CBS enzyme. It results in high levels of homocysteine in…
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