There's positive news for Evrysdi, a treatment for spinal muscular atrophy (SMA). The EMA Committee for Medicinal Products for Human Use (CHMP) has recommended that the drug be approved, and…
Sarcoma Sarcoma is a cancer that accounts for 1% of all adult cancers. It can affect individuals of any age and can impact any part of the body. It grows…
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…
In a recent study, researchers have uncovered a second gene located within the mitochondria that is associated with Charcot-Marie-Tooth Disease (CMT). This study was recently published in Annals of Neurology. CMT…
As part of ongoing events surrounding Rare Disease Day, which is officially recognized on the last day of the month of February, Patient Worthy attended sessions of the 8th Annual…
A woman living with Charcot-Marie-Tooth Disease type 2 (CMT2) has experienced symptom improvement following therapy with an anti-inflammatory compound found in green tea. This study was recently published in Medicina.…
According to GlobeNewswire, Tiziana Life Sciences and Parexel Biotech have agreed to collaborate on a Phase 1b/2 trial of foralumab, an oral treatment for Crohn's disease. As previous research has…
By: Malika Abrams I was eight years old when I found out that I was sick. After spending a beautiful summer day at the community pool with my parents, brothers,…
Ray Jordan and Cyndi Segroves were born thousands of miles away from one another; in fact, they grew up in different countries. Cyndi is from the US and Ray is…
Lysogene has just announced that the FDA has approved their Investigational New Drug Application (IND) for a therapy for GM1 gangliosidosis, a rare pediatric condition. The investigational treatment is a…
Julia's Wings Foundation is gearing up for the Operation Wear Red Campaign in an effort to raise awareness for the rare blood disorder aplastic anemia. From the first of March…
During the virtual XVIII International Conference on Duchenne and Becker Muscular Dystrophy, specialty pharmaceutical company Italfarmaco Group ("Italfarmaco") shared updates regarding various Givinostat clinical development programs. The treatment is designed…
During a recent WZDX News interview with Dr. David Bick, HudsonAlpha Faculty Investigator Dr. Bick stated that in Alabama alone, there are approximately four hundred thousand people with rare diseases.…
The Brazilian Health Regulatory Agency, ANVISA, has granted market approval to Insightec for their focused ultrasound, Exablate 4000. This MR-guided ultrasound is used for the treatment of tremors that define…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
According to a story from azfamily.com, Reyna Felix, age 28, was at work as a 911 dispatcher when her coworkers said that she started behaving strangely. Around midnight, she fell…
An estimated 1 in 10 Americans, or 1 in 20 people globally, have or will have a rare disease at some point. Rare Disease Day takes place on February 28. Ultimately,…
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