In This Issue:

🩺 Diagnosing and Managing Skin Disease in Dermatomyositis

🩺 The Utrecht JDM Cohort: Flare Prediction and the Development of an Interferon-Based Score

🩺 How JDM Impacts Family Stress and Suggested Interventions

🩺 Myositis-Associated Autoantibodies in Juvenile Myositis

🩺 The Use of JAK Inhibitors in Dermatomyositis and Juvenile Myositis

🩺 Realizing the CAR-T Potential in Myositis and Beyond

Biorepository Samples Are Available

A reminder that there are several biorepositories that store biological specimens and data used for approved research studies to researchers.  

For more information, contact Cure JM CSO Andrew Heaton, Ph.D., at andrew.heaton@curejm.org

Mental Health Grants - RFP Information

In furthering our mission of integrating mental health into pediatric rheumatology care for JM families, Cure JM is offering small grants of up to $10,000 to advance mental health care.

For inquiries regarding the RFP process and mental health grants, please reach out to Suzanne Edison, Mental Health Coordinator, Cure JM Foundation, at suzanne.edison@curejm.org

RFP Timeline:

• Application deadline August 1, 2024
• Anticipated deadline for notification of awards is October 31, 2024
• Anticipated funding begins January 2025

Research Grants - RFP Information

The Cure JM Foundation (Cure JM) announces its Request for Proposals for 2024 for advancing research to improve medical treatments, improve outcomes, and work toward a cure for Juvenile Myositis.

Cure JM is a 501(c)(3) non-profit organization established in 2003. The mission of Cure JM is to find better treatments and a cure for Juvenile Myositis and improve the lives of families affected by JM. Approximately 2 to 4 children in a million in the United States are diagnosed with JM each year.

RFP Timeline:

• Application deadline July 30, 2024
• Anticipated deadline for notification of awards is October 31, 2024
• Anticipated funding begins November 1, 2024

Recently, the Cure JM Clinical Care Network hosted its second virtual symposium of 2024, "Juvenile Dermatomyositis - Advances in Clinical Care, Basic Research, and Translational Studies." In this 14th symposium, leading researchers and clinicians presented on various topics with the goal of enhancing the overall patient journey through collaboration and best practice sharing.

We are pleased to share each presentation and the outcomes below. To review the full session recap, click here.

About this session:

Victoria Werth, M.D., will discuss the identification and treatment of skin manifestations associated with dermatomyositis, including diagnostic criteria, various skin manifestations, and the latest treatment approaches and insights into the complexities of managing dermatomyositis, aiming to improve patient outcomes and quality of life.

About Dr. Werth:

Dr. Werth is a Professor of Dermatology and Medicine at the University of Pennsylvania School of Medicine and Chief of the Division of Dermatology at the Philadelphia Veterans Administration Hospital. Dr. Werth earned her medical degree from Johns Hopkins University School of Medicine in Baltimore, Maryland. Her professional achievements include being recognized for her groundbreaking research in autoimmune skin diseases, particularly in the areas of lupus and dermatomyositis. She has also published extensively in top-tier medical journals, presented her work at major scientific conferences, and received numerous awards and honors for her contributions to dermatology and autoimmune diseases.

About this session:

The Utrecht JDM Cohort represents a pioneering initiative to unravel the complex pathophysiology of JDM flares and develop predictive tools for clinical practice. Marc Jansen, M.D., Ph.D., presents groundbreaking research elucidating the role of interferons in JDM pathogenesis and introduces an innovative scoring system for flare prediction.

About Dr. Jansen:

Dr. Jansen is a pediatrician and researcher at the University Medical Center Utrecht (UMC Utrecht) and the Wilhelmina Children's Hospital. He obtained his Doctor of Medicine (MD) degree from the University of Utrecht and completed his pediatric residency at the UMC Utrecht. Jansen received his Ph.D. in Pediatrics from the University of Utrecht in 2005. He is a leading expert in pediatric nephrology, with a focus on juvenile idiopathic arthritis-associated nephritis (JIA-AN). His research has been recognized with several awards and honors, including the "Best Paper" award at the European Society for Pediatric Nephrology meeting in 2011. Dr. Jansen has published numerous papers in peer-reviewed journals, including the Journal of the American Society of Nephrology, Pediatric Nephrology, and the Journal of Clinical Hypertension.

His research interests include pediatric nephrology, particularly juvenile idiopathic arthritis-associated nephritis (JIA-AN), the development of biomarkers and prediction models for disease activity and flares in children with JIA-AN, and the investigation of novel treatments and therapies for pediatric kidney diseases. One of his notable publications is "The Utrecht JDM Cohort: Flare Prediction and the Development of an Interferon-Based Score" (2022).

About this session:

Polly Livermore, Ph.D., will share her work with parents of children and young people with myositis, where she has been learning how we could further improve their support and, in turn, benefit their child and wider family. She will share some of the results so far about designing, developing, and testing a technology intervention to help families.

About Dr. Livemore:

Dr. Livermore is a Senior Pediatric Rheumatology Nurse at Great Ormond Street, currently serving as the NIHR GOSH Biomedical Research Centre (BRC) Clinical Academic Programme Lead. In 2020, she completed her NIHR-funded Clinical Doctoral Research Fellowship (CDRF) at Great Ormond Street, focusing on the psychosocial needs of children and young people with Juvenile Dermatomyositis (JDM). She subsequently became the first nurse in the UK to receive the prestigious NIHR Advanced Clinical Academic Fellow (ACAF) award in April 2023.

Her research initiative involves designing, developing, and testing a chatbot to support parents of children and young people with rheumatic conditions. Additionally, Polly holds various leadership roles, including Chair for Health Professionals at the Pediatric Rheumatology European Society, Associate Editor for Rheumatology Advances in Practice, and Communications Lead for the Clinical Academic Research Implementation Network (CARIN). She is also actively involved in The Centre for Adolescent Rheumatology at UCL, UCLH & GOSH and the Centre for Outcomes and Experience Research in Child Health, Illness, and Disability (ORCHID).

About this session:

Matthew Sherman, MD, MHSc, will explore different types of autoantibodies associated with juvenile myositis, their diagnostic and prognostic significance, and potential implications for treatment strategies. He will also delve into recent research findings, advancements in understanding the pathogenesis of juvenile myositis, and future directions in diagnosis and management.

About Dr. Sherman:

Dr. Sherman is a physician and researcher at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health (NIH). He is a Fellow in the Clinical Fellowship Program at the NIAMS, specializing in the Muscle Disease Section.

He received his Doctor of Medicine (MD) degree from the University of Pennsylvania Perelman School of Medicine, completed his Residency in Internal Medicine at the University of California, San Francisco (UCSF), and Fellowship in Clinical Neurology at the University of California, Los Angeles (UCLA).

His research interests include neuromuscular diseases, including muscular dystrophy and peripheral neuropathy, muscle biology and physiology, clinical trials, and translational research in muscle disease.

About this session:

Hear from Dr. Stacey Tarvin about the use of JAK inhibitors in JDM and the potential efficacy of JAK inhibitors, such as tofacitinib and baricitinib, in improving muscle strength and skin symptoms in patients.

About Dr. Tarvin:

Dr. Tarvin is an Assistant Professor of Clinical Pediatrics at Riley Hospital for Children and Indiana University School of Medicine. She is the vice chair of the CARRA JDM Research Committee and Co-leader of the CARRA JDM Therapeutics group, and she is a member of the PReS JDM working party.

She leads Clinical Research efforts in her division, including PI for pharma trials, registry studies, and investigator-initiated projects. Her focus is on therapeutics, consensus treatment plans, and telehealth in JDM. She is the Chair of Cure JM’s Clinical Care Network.

About this session:

Tania Gonzalez Rivera, M.D., discusses the application of CAR-T cell therapy in treating myositis and delves into the mechanism of CAR-T therapy, its successes, challenges, and future prospects in revolutionizing autoimmune disease treatment.

About Dr. Rivera:

Dr. Rivera is a renowned physician and executive leader in the biotechnology industry. She currently serves as the Vice President of Medical Affairs at Cabaletta Bio, a company dedicated to developing novel therapies for autoimmune diseases. Dr. Rivera completed her Doctor of Medicine and Residency at the University of Puerto Rico School of Medicine and her Rheumatology Fellowship at the University of Michigan Medical School.

Her research and clinical expertise include autoimmune diseases, particularly in the areas of rheumatology and dermatology. She has published numerous papers in peer-reviewed journals on topics such as psoriasis, lupus, and skin cancer and presented research findings at major scientific conferences, including the American Academy of Dermatology and the American College of Rheumatology.

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