On May 2, Cure JM families gathered together on Zoom to kick off this year's Walk Strong Across America. Watch the video above to meet Tom and Shari Hume, the co-founders of Cure JM, hear an exciting JM research update from Jim Minow, our Executive Director, and listen to a fun musical performance.
Join the Walk Strong Across America movement! Unite with other Cure JM families and friends to raise $750,000 for Juvenile Myositis research.
Start a team and you’ll have $100 donated to your page!
You can also give here. Gifts will be matched by the Leetz Family Match.
VAMOROLONE UPDATE
Vamorolone is what our Executive Director calls the “anti-steroid steroid.” It is a drug with all of the benefits of prednisone and virtually none of the side effects. We will soon know the clinical trial results of a study testing the drug in children with Duchenne muscular dystrophy. Those results will be reviewed and hopefully approved by the FDA later this year. That will open the path for a clinical trial in children with JDM.
Cure JM is working with Duke University Children's Hospital and the makers of vamorolone, ReveraGen, on a clinical trial to test the safety and effectiveness of the drug in children with JDM.
One of the leaders of this project, Duke University's Stephen Joseph Balevic, MD, gives us an update.
Why should this project matter to our families?
JDM is a disease that significantly affects children and their families. Currently, most children with active JDM receive corticosteroid medications such as prednisolone or methylprednisolone. While these medications are very effective, they, unfortunately, may have side effects including weight gain, poor growth, and loss of bone density. However, it is possible that new types of corticosteroids such as vamorolone may help treat patients with JDM with fewer side effects; ultimately helping children feel better with fewer side effects.
What is the current vamorolone in JDM plan?
Although there is very promising data on vamorolone from animal studies, and in children with other conditions, it has not yet been studied in clinical trials with children who have JDM. As a result, we need to study whether vamorolone is safe and effective in children with JDM by conducting a clinical trial. If we find that it is safe and effective, we also need to ensure that our patients have access to the medication (including insurance coverage), which would mean applying to the US Food and Drug Administration (FDA) for drug approval. Fortunately, the FDA provides guidance to investigators who are in the early stages of clinical trials. Right now, we are in the process of designing a clinical trial to evaluate the safety of vamorolone and help identify the doses needed to control disease in JDM. We plan to meet with the FDA soon to ensure that the study has the best design possible.
RECORDING OF “THE IMPORTANCE OF EXERCISE IN JUVENILE MYOSITIS” TOWN HALL
This Town Hall explored the importance of different types of exercise in the management of JM and was presented by Sue Maillard, a specialist physiotherapist at Great Ormond Street Hospital, London.
Sue has completed research in the safety of exercise in JDM and leads a physiotherapy team to provide an inpatient rehabilitation program for children, as well as reviewing and managing home treatment programs.
