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…
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