Archive for the Disease Awareness 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

IT’S RARE DISEASE DAY AND WE ARE ADVANCING CTD KNOWLEDGE!

By Carol A. Dutch – Senior Director, Patient Engagement, Lumos Pharma More than two years ago, a multidisciplinary team of investigators based primarily at Children’s Hospital of Philadelphia (CHOP) set out to advance the scientific and medical community’s understanding of a rare pediatric neurodevelopmental disorder called Creatine Transporter Deficiency (CTD/SLC6A8), first identified in 2001. Their work culminates...Read More

Two new Vigilan sites open at Duke and Rush

Duke University Medical Center in Durham, North Carolina and Rush University Medical Center in Chicago, Illinois are now recruiting individuals to participate in a two-year prospective observational study of males with Creatine Transporter Deficiency (CTD), named the Vigilan Study. Dwight Koerberl, MD, PhD is the principal investigator (PI) at Duke University, and Elizabeth Berry-Kravis, MD,...Read More

Lumos Pharma launches ScreenCreatine.org Disease Awareness Website

Austin, Texas – March 15, 2017 ScreenCreatine Project: Disease awareness website launched to expand understanding and resources associated with Cerebral Creatine Deficiency Syndromes (CCDS) including Creatine Transporter Deficiency (CTD) Today, Lumos Pharma launches the disease awareness website ScreenCreatine.org, part of the ScreenCreatine Project.  ScreenCreatine.org was developed to provide health literate information about the three creatine...Read More