Archive for the Orphan Drug News Category

Transferring Sponsorship of a Clinical Study: A lesson on how rare disease drug developers can work together for the good of an underserved rare disease community

    On June 20th, 2019, wonderful news was announced on the Association for Creatine Deficiencies’ (ACD) website and ACD Facebook page that Lumos Pharma has transferred the sponsorship role of the Vigilan Study (Observational Study of Creatine Transporter Deficiency-CTD) to Ultragenyx Pharmaceutical Company. https://creatineinfo.org/wp-content/uploads/2019/06/Vigilan-Update-June-20-2019-Transfer-ACD-Letter.pdf “The ongoing natural history study, Vigilan, is crucial for the...Read More

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