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When a child is diagnosed with juvenile myositis, families quickly learn how rare and complicated this disease can be. They also ask one of the most important questions any parent can ask:
Is my child’s doctor fully knowledgeable on the latest research, treatment options, and care recommendations?
At Cure JM, one of our most important responsibilities is helping make sure the answer to that question is an unqualified YES!
Juvenile myositis is rare. Many physicians will see only a small number of patients in their careers, and even excellent pediatric specialists may have limited experience with the disease. That is why Cure JM has invested more than two decades building a network of physicians, researchers, families, and supporters working together to improve outcomes for children with JM.
That network is especially important now.
The JM medical field is moving quickly. Researchers are learning more about how to treat the disease earlier, define clearer treatment goals, prevent long-term complications, and study new therapies that will and already has changed the future for children with JM.
Some of this progress is happening in the United States. Some is happening in Europe. Some is happening through global collaboration among experts focused on better treatments and better outcomes.
Cure JM’s role is to help make sure that knowledge does not remain limited to one publication, meeting, or institution. We help move it into the hands of the doctors caring for children.
One example is CAR-T cell therapy. CAR-T was first developed as a cancer therapy, but researchers are now studying whether it can help reset the immune system in certain severe autoimmune diseases. In juvenile myositis, early case work from Dr. Rebecca Nicolai and her colleagues at Bambino Gesù Children’s Hospital in Rome has helped bring new attention to this field.
CAR-T is not yet a standard treatment for JM, but it is well on its way. CAR-T is one of the most important research frontiers in autoimmune disease, and Cure JM is helping ensure that JM families and physicians are included as this science advances.
Through the RESET-Myositis clinical trial led by Cure JM’s industry partner, Cabaletta Bio, children and young adults with juvenile myositis are now beginning to access a research opportunity that was not available just a few years ago. Many Cure JM Clinical Care Network children’s hospitals are participating, and Cure JM is helping support the education, awareness, and clinical trial readiness needed for families and clinicians to understand these opportunities.
This is where Cure JM’s network matters.
We connect families to information. We connect doctors to experts. We connect researchers to the patient community. And we help prepare the JM field for the next generation of clinical trials.
CAR-T is not the only area of progress. Researchers are also studying more targeted therapies, including JAK inhibitors and other emerging approaches that may help children with JM achieve better outcomes with fewer long-term complications. Across the field, there is growing focus on earlier intervention, clearer treatment goals, and more precise care.
The key takeaway for families is simple:
Cure JM is working to make sure the latest knowledge reaches the doctors caring for children with JM.
That happens through physician education, research and clinical summits, Clinical Care Network updates, family Town Halls, clinical trial readiness efforts, and direct communication with the families who need answers most.
Cure JM has invested more than $32 million in research, physician education, clinical care initiatives, and mission programs that move the field forward. Those investments are helping build the foundation for today’s momentum.
This progress is encouraging, but it is not automatic.
Clinical trials require prepared hospitals, trained physicians, informed families, outreach, and support. Pediatric rheumatology remains significantly under-resourced, and rare pediatric diseases like JM often do not receive the level of institutional investment needed to move promising work forward.
That is why Cure JM’s role matters. Making a gift to our Clinical Research Fund helps prepare the field so that when research opportunities emerge, the JM community is ready to understand them and move them forward responsibly. Our summer Walk Strong Across America campaign helps amplify that same mission by raising awareness and strengthening the community behind these efforts.
The field is changing rapidly, and Cure JM will continue helping families and clinicians stay connected to that progress.
Thank you for standing with Cure JM and helping move this work forward.
With gratitude,
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