Rare Disease Day at NIH: Finding Hope in Gene Therapy
The National Institutes of Health (NIH) held its annual Rare Disease Day event on March 1, 2021 this year. This event showcases groundbreaking research, amazing rare patient stories, and more.…
The National Institutes of Health (NIH) held its annual Rare Disease Day event on March 1, 2021 this year. This event showcases groundbreaking research, amazing rare patient stories, and more.…
The FDA has just announced their approval of Evkeeza as a therapy for those living with homozygous familial hypercholesterolemia. The therapy is owned by Regeneron Pharmaceuticals. It was evaluated with…
Pharnext has announced that they will soon open enrollment for their PREMIER trial of PXT3003, a Charcot-Marie-Tooth disease type 1A (CMT1A) treatment. Beginning in March, researchers will search for 350…
There is a new collaboration in the rare cancer space which could lead to improved therapies for individuals living with these conditions. Sheperd Therapeutics and Oncoheroes Biosciences have announced that…
Researchers from the Universities of Nottingham and Exeter have discovered a new, possible treatment method for Duchenne muscular dystrophy (DMD). Their research demonstrated that treating hydrogen sulfide (H2S) deficiency in…
It's been almost a full year since the beginning of the COVID-19 pandemic, and we've learned a lot about the novel coronavirus since then. For one, we now know that…
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…
Crispr is no longer a buzzword. According to a recent article in The Guardian, it has become the ‘molecular scissors’ that will enable scientists to rewrite our genes or as often said,…
According to a recent press release, neuropharmaceutical company NeurAegis Inc. shared the publication of some study data which explored calpain-2 as a potential therapeutic target for status epilepticus (SE). The…