According to a story from the AKU Society, the European Medicines Agency (EMA) has recently announced a positive opinion on extending the label indication for nitisinone (marketed as Orfadin®). The…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Let me begin by saying that…
The Value of a Service Dog September has been deemed National Service Dog Month. These animals are essential for many people living with chronic conditions, disabilities, and various rare diseases.…
A study reported in NewsWise evaluates retinoid-x receptor (RXR) agonists as a treatment for recurring and refractory multiple sclerosis (MS). This specific RXR agonist, bexarotene, was found to help remyelination,…
aHUS Tracking Atypical hemolytic uremic syndrome (aHUS) is a rare disease that causes blood clots to form within the small blood vessels in the kidneys. These clots lead to organ damage…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
According to an article from BioSpace, Vivet Therapeutics and Pfizer have entered into a manufacturing agreement to develop Vivet's gene therapy, which treats Wilson disease. The terms of the agreement…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Dave Kinler and I have walked…
Damian Green from the University of Washington is the Principal Investigator for a new research project in multiple myeloma. This project is thanks to a research agreement between the Fred…
The question of whether to go back to school amidst Covid-19 has been a debate across the country. Many universities have seen surges in cases, many involving students in Greek…
Dr. Lisa Sammaritano from Weill Cornell Medical College has explained a way to lower risks within pregnancy for those with rheumatic diseases. Pregnancy and Rheumatic Diseases Patients who have a…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson reported that they submitted a New Drug Application (NDA) for intravenously administered UPTRAVI (selexipag). The therapy is designed to treat…
Acknowledgment: This story is sponsored by Chiasma, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
Vertex Pharmaceuticals will present data on the long-term use of their cystic fibrosis (CF) treatments at the 43rd European Cystic Fibrosis Digital Conference, and then again at the 2020 North…
APT-1011 is a treatment for eosinophilic esophagitis, a condition that currently faces an unmet medical need. The medication was originally developed by TPG Capital, but they created a new company,…
Recently, twenty-two year old Jordan Ray of Wellington, Florida spoke with a reporter from The Palm Beach Post, recounting how her life changed in April 2015 during a softball…
by Danielle Bradshaw from In The Cloud Copy There was a case report (called Anti-MUSK positive myasthenia gravis with antiLrp-4 and antigen antibodies) released recently that talked about a patient…
Researchers have announced their identification of a specific gene mutation that can lead to atypical hemolytic uremic syndrome (aHUS). This gene is called DGKE and it is not a part of…
A research team at Ghent University in collaboration with Imec recently released preliminary data on their artificial iris. They are calling it a "smart contact lens." By embedding microelectronics into…
Misophonia is a rare condition that causes patients to have a severe and involuntary reaction to specific sounds. The cause of the condition is still unknown. A challenge misophonia patients face…
Disclaimer: This blog post has been republished with permission from the author. To visit the original blog site, click here. Written by Tamara Fowler Today was the end of the…
Immune-One Therapeutics has just announced that they have dosed their first acute myeloid leukemia (AML) patient in their Phase 1 trial investigating IO-202. This therapy is an immune inhibitory receptor that works…
by Danielle Bradshaw from In The Cloud Copy Research into the common but rare TP53 mutation - which can be found in many Ashkenazi Jewish people - revealed that it…
Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…
According to an article from Charcot-Marie-Tooth News, a study was conducted that discovered three new mutations on the GJB1 gene that result in X-linked Charcot-Marie-Tooth disease (CMTX1). The study, which…
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