Cure JM April 2022 Family News

CURE JM MAY 2022 FAMILY NEWS

Our Mission: To find a cure and better treatments for Juvenile Myositis and improve the lives of families affected by JM.

CONTENTS

Walk Strong Across America

UCSF Benioff Children's Hospital Named Center of Excellence

Family Spotlight

CARRA Telemedicine Survey

Key Milestone for Possible Clinical Trial for Vamorolone

Upcoming May Town Hall

Top 10 Takeaways from the Cure JM Town Hall: Pain, Mental Health and JM

DONATE

Third Annual Walk Strong Across America on July 4th, 2022

Cure JM is pleased to announce our 3rd annual Walk Strong Across America. Cure JM families, friends, and supporters from across the country are rallying to raise $750,000 for JM research by July 4th.

Your family can start a team today. We invite you to walk from the comfort of your home, in your yard, or around your neighborhood. With your support, we can pursue new treatments, fund clinical trials, expand our Centers of Excellence, and expand our NEW Cure JM Clinical Care Network, which means more excellent care to children around the country.

Ready to start your team? Contact Lauren Alvord at Lauren.Alvord@CureJM.org or click on the button below to get involved in Walk Strong.

If you would like to plan a kick-off event for Walk Strong in your area, contact Betsy Leon, Betsy.Leon@CureJM.org. There are currently 9 Cure JM in-person Chapter events already planned.

Meet the Walk Strong Co-Chairs:

Kristine Alderfer and Karen Berman

Kristine has served as president of the Cure JM Board of Directors since 2021, and has been an advocate for Cure JM since 2010. Kristine and her husband Alan have twin daughters, Katherine and Caroline. Katherine was diagnosed with JM when she was four years old.

In 2013, Kristine was named "Cure JM Hero" for her outstanding efforts. Kristine is the National Co-Chair of the Walk Strong Across America. Kristine is the National Co-Chair of the Walk Strong Across America.

Karen started volunteering with Cure JM in 2016 after her daughter was diagnosed with JDM. Karen was a fundraising coach for the 2017 and 2018 Chicago Chapter Walk Strong events. In 2019, she received the Cure JM Hero Award for exceptional service as the Chairperson of the Chicago Walk Strong to Cure JM, which raised $135,000. Since then,

Karen has served as the national Co-Chair for the Walk Strong Across America, leading the charge for Cure JM's signature fundraising event.

Learn More About Walk Strong Across America

UCSF Benioff's Children's Hospital San Francisco Named Cure JM Center of Excellence

The Cure JM Foundation recently named UCSF Benioff Children's Hospital San Francisco (UCSF) a Center of Excellence. Cure JM Centers of Excellence are recognized for the extraordinary care they provide to Juvenile Myositis patients and families.

The dedication of the clinicians at UCSF to treating patients with JM is truly formidable. Susan Kim, MD, MMSc is an Associate Professor and Associate Clinical Professor of Pediatrics at UCSF. She is a Board Certified Pediatric Rheumatologist who joined the faculty at UCSF in 2016. She specializes in caring for children and adolescents with inflammatory rheumatic diseases, including Juvenile Dermatomyositis.

Jessica Neely, MD is a Pediatric Rheumatologist, as well as an Assistant Professor of Pediatrics at UCSF. She diagnoses and treats children with all types of autoimmune diseases and is also a clinical and translational researcher.

"We are honored to be chosen as a Cure JM Center of Excellence at UCSF. In partnership with our patients and families, we are excited to align clinical care and research to improve the outcomes of patients with JDM today, as we build toward a future that is better and brighter for all children affected by JDM," Dr. Kim and Dr. Neely jointly said.

Learn about all of the Cure JM Centers of Excellence at curejm.org/centersofexcellence

FAMILY SPOTLIGHT

Caroline's Infusion Day is available on Amazon.com

Recently, the Chicago Tribune featured an article about Cure JM Mom, Kelly Brennan and her book, Caroline's Infusion Day. The article discusses how the book evolved, mainly out of a lack of stories regarding infusions, and Kelly decided, "We need to have a book for this."

Caroline's story is a lot like other JM patients, where spending hours of time receiving IV medication becomes a routine. As stated in the article, for the first two years of treatment, Caroline was going in for infusions every four weeks. For many JM families, after diagnosis and starting treatments can be very isolating. This is exactly why Kelly wrote the book--to let families know that you are not alone.

Caroline was diagnosed at 2, and now 4, she is still receiving treatments, but she is a happy, spunky little girl. Recently, her family went to Disneyworld for vacation, and she loved every minute.

As Kelly notes, "I don't know why this is her journey, but I know great things come from challenges. Caroline will not let this disease limit her."

Caroline's Infusion Day has helped many other JM families and patients deal with pokes and days of treatment, and Kelly is glad for that. "My next plan is to start a Podcast for JM families, and I am hoping that serves as a source of support, as well as education about JM."

Stay tuned for more to come from Kelly Brennan!

CARRA Telemedicine Survey

The JDM CARRA telemedicine workgroup has been hard at work on trying to come up with standard practices for telemedicine appointments with our JM kids.

Please consider taking this survey to offer your feedback. Thank you.

CARRA SURVEY

Research Update: Key Milestone for Possible Clinical Trial for Vamorolone

Andrew Heaton, PhD, Chief Scientific Officer, Cure JM

There is an exciting update to share about the drug vamorolone, a possible new, better treatment for JM.

Through a partnership with Duke University/Duke Clinical Research Institute, the pharmaceutical company Reveragen, and Cure JM, we have moved forward in the process toward a potential clinical trial of vamorolone with JM patients.

A key milestone has recently been achieved through the submission of a pre-Investigational New Drug (pre-IND) meeting request to the FDA to discuss the design of the trial and work towards initiating the study.
In the past, the FDA has always moved carefully with drug trials involving treatment of a rare disease to ensure safety. This pre-IND meeting will help to move the discussion forward.
The pre-IND meeting should happen within the next 3 months.

This is exciting progress, and updates will continue to be shared with you as we know more.

UPCOMING EVENT

May 13, 2022 Town Hall

Time: Noon Pacific/1PM Mountain/2PM Central/3PM Eastern

Myositis Specific Antibodies: What Families Need to Know

Presenters: Jessica Neely, MD and Chair of the Cure JM Board of Directors and Cure JM Mom, Nikki Hahn

Myositis-specific antibodies (MSAs) have been shown to be associated with certain symptoms in JM patients. In some cases, knowing the MSA for a patient, may help clinicians look out for certain symptoms and also guide treatment decisions. Dr. Neely will discuss these associations and why they are important as well as the limitations that still exist in our understanding of the role of MSAs in JM. Nikki Hahn will offer her perspective as a JM Mom about MSAs.