|
Cure JM is leading a new era in which emerging therapies are being designed around a deep understanding of the pathways that drive myositis. Brepocitinib, a new drug from Priovant Therapeutics, joins other promising approaches, including cell therapies such as CAR-T, that would have seemed extraordinarily distant only a few years ago.
Priovant is currently seeking FDA approval for brepocitinib, which is a once-daily oral therapy studied in adults with dermatomyositis. The FDA has accepted the application with Priority Review, following positive results from a Phase 3 clinical trial. If approved, possibly in the next few weeks, brepocitinib would become the first targeted therapy approved specifically for dermatomyositis.
Brepocitinib inhibits both JAK1 and TYK2, two proteins that help transmit inflammatory signals through the immune system. These pathways include interferon-related signaling, which researchers have increasingly linked to the biology of dermatomyositis. In simple terms, rather than broadly suppressing the immune system, therapies like brepocitinib are designed to interrupt more specific signals driving inflammation.
For almost 25 years, Cure JM has been creating the clinical and research infrastructure that allows promising treatments to move forward.
Through your support, Cure JM has invested in leading physicians, research institutions, Centers of Excellence, the Clinical Care Network, clinician education, and clinical-trial readiness. Those investments help ensure that when promising new therapies reach the clinical-trial stage in pediatrics, there are experienced physicians, prepared institutions, and an informed patient community ready to participate.
That infrastructure matters more today than ever as scientific discovery accelerates.
Our responsibility is to ensure that the juvenile myositis community is ready as pediatric trials are underway.
I also want to take a moment to recognize and important scientific contribution from a long-standing member of the Cure JM Board of Directors, Dr. Tracy Van Ness. Dr. Van Ness recently published the peer-reviewed article Juvenile Dermatomyositis: Multisystem Impacts and Evolving Treatment Paradigms.
Published in the 2026 edition of Physician Assistant Clinics, the article provides a clinically focused overview of juvenile dermatomyositis, including its effects across multiple body systems and the evolving approaches used to understand and treat the disease.
As JM mom, Tracy brings a distinctive and deeply valuable perspective to the JM community. She is a healthcare professional with more than two decades of clinical experience, but she also understands JM from the perspective of a parent. Today, she brings both perspectives to her service on Cure JM's Board of Directors and to her work on behalf of our community.
Her publication is another example of the growing body of knowledge surrounding juvenile myositis and of the extraordinary expertise that exists within the Cure JM community itself.
Access the Full Publication
Both of these stories reflect something I find deeply encouraging about where we are today. The science is moving forward, our understanding of JM continues to deepen, and the Cure JM community is helping build the knowledge, partnerships, and infrastructure needed to turn that progress into better outcomes for children.
I am grateful to everyone who continues to help Cure JM move that progress forward. Your support of the Clinical Trial Fund helps ensure that the physicians, institutions, and families needed to advance promising new therapies are ready when the opportunity comes.
| 
