Archive for the Orphan Drug News Category
10 CTD Study Sites Now Open
The Vigilan Study – Observational Study of Creatine Transporter Deficiency (CTD) of Males, now has ten study sites open for patient enrollment. Males with CTD will be screened at university clinics. Once enrolled in the study, participants will undergo a series of age-appropriate tests of physical and intellectual abilities. Follow-up testing will occur every 6...Read More
Lumos Pharma Celebrates Worldwide Rare Disease Day with “Screen Creatine NOW!” Video
One in ten individuals are living with a rare disease. On the last day of February every year, hundreds of patient organizations from more than 80 countries conduct awareness-raising activities to celebrate Rare Disease Day. In honor of the 10th annual Rare Disease Day, Lumos Pharma has created the below video “Screen Creatine NOW!” in...Read More
National Institutes of Health Creates Six New Projects as Part of Therapeutics for Rare and Neglected Diseases (TRND) program.
AUSTIN, Texas – July 12th, 2014 Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease. The six...Read More