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Juvenile Dermatomyositis: Advances in Basic Research, Translational Studies and Clinical Care

May 13, 2023, at 10:00 a.m. EST

3.0 CME credits are available through Duke University

Session 1

10:00 – 10:10:

Opening Comments and Introduction. Nikki Hahn (Chair, Cure JM Foundation Board of Directors), Andrew Heaton Ph.D. (CSO, Cure JM Foundation)

10:10 – 10:35:

Sarah Tansley, MBChB, PhD (Bath, UK)

Current Position:  Senior Clinical Lecturer and Honorary Consultant Rheumatologist

Main area of research focus:  Identification and clinical associations of autoantibodies in rheumatic disease, particularly myositis

Myositis Autoantibody testing in JM; practicalities and pitfalls

10:35 – 11:00:

Andy Mammen, MD, PhD (NIH, USA)

Current Position: Senior Investigator, Muscle Disease Section, NIAMS/NIH

Main area of research focus: Autoimmune muscle diseases

The clinical importance of autoantibodies in juvenile myositis

11:00 – 11:25:

Jessica Neely, MD (UCSF, USA)

Current Position: Assistant Professor at the University of California, San Francisco

Main area of research focus: To better understand immune dysregulation in JDM as well as identify biomarkers and therapeutic targets using multi-modal -omics and integrative computational approaches.  

Dr. Neely is also the co-director of the Cure JM Center of Excellence at UCSF and directs the Juvenile Myositis Precision Medicine Cohort and Biorepository at UCSF.  She is also the Co-Leader of the Translational Medicine for Juvenile Myositis CARRA Working Group.  
Immune Dysregulation in JDM at Cellular Resolution

11:25 – 11:50:

Jessica Turnier, MD (University of Michigan, USA)

Current Position: Assistant Professor of Pediatric Rheumatology at the University of Michigan.

Main area of research focus: Understanding how cutaneous inflammation influences systemic disease development, disease progression and disease phenotypes in juvenile myositis, with an overarching goal to identify novel biomarkers and therapeutic targets to develop precision medicine care for juvenile myositis patients.

Dr. Turnier is part of Cure JM Clinical Care Network, coordinating PI for CZI grant for which we are partnered with Cure JM, development of JM CAB in conjunction with Cure JM

Implementing the use of nailfold capillaroscopy in juvenile myositis clinical care and research

11:50 – 12:15:

Rohit Aggarwal, MD, MS (University of Pittsburgh, USA)

Current Position: Professor of Medicine at University of Pittsburgh and Medical Director of Arthritis and Autoimmunity Center at University of Pittsburgh Medical Center. He is the co-director of UPMC Myositis Center

Main area of research focus:  A leader in disease criteria and outcomes measures in myositis as well as in clinical trials of novel therapeutic agents for myositis He continues to be a leader in disease criteria and outcomes measures in myositis as well as in clinical trials of novel therapeutic agents for myositis

The ProDERM study: Safety and Tolerability Results from a randomized, double-blind, placebo-controlled Phase-III-Trial in Patients with Dermatomyositis

Session 2

12:15 – 12:40:

Keynote Speaker Lisa Rider, MD (NIEHS, NIH, USA)

Current Position: Head, Environmental Autoimmunity Group, National Institute of Environmental Health Sciences (NIEHS), National Institutes of Health (NIH).

Main area of research focus:  Myositis in children and adults, environmental and genetic risk factors, outcome assessment, myositis autoantibodies, therapeutic trials.

Dr Rider is  Chair, Cure JM Medical Advisory Board;  Stanley Manne Children’s Research Institute/ Ann & Robert H. Lurie Children’s Hospital of Chicago Cure Juvenile Myositis Center of Excellence Biorepository and Registry Review Committee; NIEHS liaison/representative  to NIH Office of Autoimmune   Disease Research and NIH Coordinating Committee for Autoimmune Disease Research, Unpaid Medical Advisor to Pharmaceutical and Biotechnology companies (current ones include Horizon Therapeutics, CSL Behring, Pfizer) 

Abatacept for Juvenile Dermatomyositis and Adult Myositis: A Tale of Discordant Trials

12:40 – 13:05:

Christian Lood, PhD (University of Washington)

Current Position: Associate Professor Division of Rheumatology, Department of Medicine, University of Washington, Seattle, WA, USA.

Main area of research focus: Mitochondrial-mediated inflammation, autoimmunity and organ damage.

Dr. Lood is part of the steering committee Seattle Children’s Hospital Cure JM Center of Excellence

Mitochondria in JDM

13:05 – 13:30:

Hanna Kim, MD (NIH, USA)

Main area of research focus: In addition to detailed research evaluations of juvenile myositis patients at NIH, she also leads clinical trials of novel treatments in clinical trials in juvenile myositis including leading a previous study with baricitinib (janus kinase inhibitor).

Janus Kinase inhibitors in JDM

13:30 – 13:55:

Andrea Knight, MD, MSCE (Hospital of SickKids Toronto, Canada)

Current Position: Pediatric Rheumatologist, The Hospital for Sick Children.

Associate Scientist, SickKids Research Institute, Neurosciences and Mental Health Program. Associate Professor of Paediatrics, Faculty of Medicine, University of Toronto. Canada Research Chair in Mental Health and Chronic Disease of Childhood.

Main area of research focus: Dr. Knight’s research program focuses on mental health in youth with rheumatologic diseases such as lupus and juvenile myositis. She has investigated the burden of psychiatric morbidity, as well as strategies to improve comprehensive care for these patients. She is also investigating the impact of lupus on brain structure, function and development

Dr. Knight has many roles in JM including Co-leader of the Mental Health Workgroup for the Childhood Arthritis & Rheumatology Research Alliance (CARRA)

Moving the needle: Improving Mental Health for Children with Juvenile Myositis

13:55 – 14:05:

Closing Remarks. Jeffrey Dvergsten, MD (Duke University)

Affordable and Accessible Treatments for JM

Affordable and Accessible Treatments for JM

Two special guest speakers, Michelle Vogel, MPA, IV Solutions RX, and Laurel Cherwin, BSN, RN, IgCN, Octapharma, shared information on navigating affordable treatments and care for JM patients.

The FDA’s Ruling on Vamorolone in DMD

You may have heard about yesterday’s FDA ruling, which approved vamorolone as an alternative steroid for use in Duchenne muscular dystrophy.

Vamorolone is a new steroid alternative with fewer side effects than prednisone. The drug aims to preserve the beneficial anti-inflammatory and muscle-strengthening characteristics of corticosteroids, while decreasing some of the unwelcome side effects, including bone fragility, delayed growth, and behavior changes.

As we learned in yesterday’s announcement, vamorolone was found to be both safe and effective as a therapy for Duchenne muscular dystrophy in its newly approved indication.

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