Dear Cure JM Community,

I am excited to report that in recent months Cure JM’s quest—our mission to discover better treatments and a cure—is closer than ever to realization.

You may have heard by now that a new drug therapy, now known as Lisraya, was approved by the FDA for use myositis in August and will soon be available by prescription for DM patients 18 and over. This is the first drug ever to be approved in myositis, and importantly, we are speeding toward submitting additional data to the FDA that we are confident will result in Lisraya’s approval for use in children.

Clinical results from Lisraya are stunning: patients in the trial were able to discontinue steroid use and saw dramatic increases in Total Improvement Score, the key measurement improvements in muscle strength, skin, joint, and lung improvement, and overall disease activity.

Dr. Ruth Ann Vleugels, who presented these results to our community on September 24, summed up the promise of this moment. She explained that newer therapies like Lisraya should not be held in reserve, especially when we know older treatments such as steroids and methotrexate carry damaging side effects and don't always work. Lisraya blocks pathways that help drive myositis, a more targeted approach than simply suppressing the immune system.

Dr. Vleugels is the recently appointed chair of the Department of Dermatology at the University of Pennsylvania and serves on the Board of Directors of the American Academy of Dermatology. This month, we welcome Dr. Vleugels to the Cure JM Medical Advisory Board.

I would be totally remiss if I failed to provide you with an update on another clinical trial that may well prove to be curative—CAR-T therapy that resets a child’s entire immune system. The pediatric arm of this trial is fully enrolled with patients as young as six years old. Again, the early patient-reported outcomes are astounding.

Patient-reported outcomes are indeed important, but what will matter most to the FDA is the data collected in the trial from a larger number of U.S. patients. In short—this clinical trial would not have been possible without the active engagement and leadership from Cure JM families. One of our earliest trial participants is now reporting that her teenage daughter who has been battling JM since early childhood is now in CAR-T remission.

Without you, and without our network of Centers of Excellence and children’s hospitals, this trial in children would not have been possible. Through your support, Cure JM has invested heavily in the development of pediatric rheumatology research and training through our children’s hospital network—the very doctors now serving as Principal Investigators driving toward the approval of CAR-T in children.

This week our families across the country launch the most important fundraiser of the year—the Cure JM Holiday Challenge. Year in and year out, the Holiday Challenge sets the pace for everything Cure JM does or will ever do to advance the research and support the doctors and children’s hospitals providing the best possible care to JM children everywhere.

There has never been a more hopeful time to join the Holiday Challenge. An ever-

increasing number of clinical trials is speeding us toward better treatments and a cure, and Cure JM is working to make sure children are part of every one of them. We bring together the doctors, hospitals, and families that pediatric trials depend on. We sit at the table with researchers and pharmaceutical partners, and we make sure one thing is never forgotten: children with JM cannot be an afterthought.

But momentum alone does not reach a child. Funding does. Every fundraiser and every gift fuels research, prepares our hospitals to take part in studies, and brings new treatments closer to the kids and families who are waiting for them.

This holiday season, sign up for the Holiday Challenge and invite your friends and

family to rally behind you. And if you can, make a gift of your own today at

www.curejm.org/acurestartswithyou. Together, we can turn this progress into a cure for every child living with JM.

A cure starts with you.

With gratitude,