Cure JM Foundation

Patient-Partnered Collaboration
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Juvenile myositis involves a run-away immune response where the body’s immune system attacks its own cells and tissues. With proper treatment and care, children can go on to live their best lives.

Last updated May 2024

Clinical
Disease Class
Rare immunological diseases
Body Systems
Hematopoietic / Lymphatic / Immune
Muscular / Skeletal
Organs
Connective tissue / joints
Lungs
Muscles
Skin
Genes
None specified / unknown
Type of Inheritance
Not specified / unknown
Disease Mechanism(s)
Autoimmune
Age of Onset
Middle childhood (6-11)
Incidence
Less than 10
Prevalence
101-1000
Populations and/or ancestry with higher prevalence
Females are affected more frequently than males (2-5:1 ratio)
Symptoms / Phenotypes
inflammation
muscle weakness
myositis
skin redness and/or swelling
Biomarkers
Diagnostic
· Myositis Specific Autoantibodies
Monitoring
· IFN signature genes
Prognostic
· Myositis Specific Autoantibodies
Existing Therapies
Alternative treatments (eg. nutritional supplements)
· physical therapy
Drugs used off-label
· corticosteroids, mycophenolate mofetil or biologic medications such as rituximab, tofacitinib, abatacept, adalimumab and infliximab.
Organizational & Research
Cell Lines
B lymphocytes
Myoblasts
Plasma
Cell Lines, location
Not specified
Cell Lines, share
Unknown
Disease Model
Mouse
Disease Model, location
SUNY Binghamton
Disease Model, share
Yes
Clinical Trial Role
Focus group
Funding
Meeting with regulators
Recruitment and outreach, patients
Recruitment and outreach, trial sites/physicians
Results dissemination, publication
Biobank
Lurie Childrens Hospital of Chicago
Center of Excellence
Duke University
George Washington University
Seattle Children's Hospital
University of California San Francisco (UCSF)
Registry
Yes, we have collaborated on a registry
Data Collected, Registry
Clinical data
Longitudinal natural history data
Medication usage
Patient contact info
Patient-reported data
Data Entered by, Registry
Clinicians
Platform, Registry
REDCap
Natural History Study
Yes, we have collaborated on a natural history study
Data Collected, Natural History Study
Patient-reported outcomes
Retrospective data
Platform, Natural History Study
REDCap
FDA Patient Listening Session
Yes
FDA Patient-Focused Drug Development (PFDD) Program
No
ICD Codes
Yes, we have an ICD-11 code specific to our exact disease
Diagnostic Guidelines
Yes
Clinical/Treatment Guidelines
Yes
Organizational Roles
Executive Director
Patient Engagement Manager/Director
Research/Scientific Director
Science Advisory Board Policies
Yes, willing to share SAB policies
Research Network Policies
Has CRN and willing to share policies
Research Roadmap
Yes we have a Research Roadmap, and will share policies
International Chapters
None
International Partners
Europe
North America