If you're worried or anxious because of COVID-19, you're not alone. If wondering about the intersection of rare diseases and COVID-19 only increases that anxiety, well, we're with you…
Cabozantinib has recently been studied as a treatment for Ewing sarcoma and osteosarcoma. It demonstrated antitumor activity in both of these cancers, which was discovered in the second phase of…
Crohn's disease is a very rare condition in Taiwan, as only .2 of every 100,000 people are diagnosed with it. Because of this rarity, there is little to no awareness…
According to a story from globenewswire.com, the biopharmaceutical company Liquidia Technologies, Inc. just announced that the company's New Drug Application (NDA) has been officially accepted by the US Food and…
According to a recent article in Parkinson’s News Today, the drug prasinezumab has entered the second half of a Phase 2 trial. An earlier trial, NCT02157714, showed that the therapy…
As originally reported in Biospace, Cushing disease, a disease due to excessive cortisol, has recently had its first ever treatment option approved by the FDA for adult patients who are…
According to a story from Biospace, in Palo Alto, AI company Emedgene plans to dive into helping the undiagnosed with their new rare disease initiative. The company will be paired…
COVID-19 and its rapid global spread have been anxiety-inducing, to say the least. For patients with rare diseases and their families, the growth of COVID-19 has also prompted additional…
Rare, neurologic diseases often do not have viable treatments. Gaucher disease and Parkinson's disease are two examples. In situations like these, medical professionals look to treatments for other conditions, such…
Shannon and Todd Palmatier have three children, two of which have progressive familial intrahepatic cholestasis (PFIC). Their sons' diagnoses have brought the lack of knowledge and advocacy to the attention…
Social Distancing Social distancing has been coined as the most surefire way to stop the spread of COVID-19 across the American populace. What is this phrase used to refer to?…
No one was given a prep course on how to handle quarantine, especially not with kids. Here are some suggestions to help you get through these unique times, while making…
As Patient Worthy readers, we hope you and your families are doing well during this challenging time. The health and safety of our rare community is always at the…
A news release from Benzinga shares that biotechnology company Emerald Health Pharmaceuticals is beginning a Phase 2 clinical study on EHP-101 as a treatment for patients with systemic sclerosis.…
Every day on the news we hear the pleas for more medical masks for doctors and nurses, more personal protective equipment, and more ventilators. We all know what masks are,…
According to a study published in the journal Neurology, a measure called the vastus lateralis fat fraction was found to be useful in predicting the age at which a patient living…
Researchers at Columbia University have discovered a new gene, TBK1, that is linked to the progression of amyotrophic lateral sclerosis (ALS). A lack of understanding of a rare disease hinders…
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…
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…
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…
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…
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