The Importance of Research

Researcher discusses chart on wall.

“There is one reason my daughter is alive and close to remission today,” a JM mom recently wrote to Cure JM in a letter of thanks. “That reason is research.”

We could not have said it better ourselves.

When Cure JM was founded 20 years ago, little was known about juvenile myositis—what caused it or how to treat it effectively. There were few experts among pediatric rheumatologists, and because JM is a rare disease many doctors and pediatricians had not heard of, its symptoms were often misdiagnosed and treatments delayed.

Before researchers discovered that corticosteroids could effectively suppress an overactive immune response in JM, one in four affected children would die from the disease. Today, the mortality rate is below 3%. This is why research is so important. What we learn—what doctors learn—from Cure JM-funded research has saved lives, improved treatments, and increased access to quality experienced care throughout the U.S. and worldwide.

Today, because of our research, we know more about how to treat this diverse and complex disease than ever before. We know, for example, that children have different autoantibodies that can often tell us something about the course of the disease and how it might affect muscles, skin and other organs in the body, such as the heart and lungs.

We now have standard measurements to evaluate lab results, which has led to the creation of widely-used treatment protocols with more predictable outcomes, reducing the guesswork around clinical care.

Through training and physician education, more doctors than ever before can provide experienced, quality care for your JM child.

Cure JM is laser-focused on developing new or repurposed drugs that will be more effective than corticosteroids in treating JM without debilitating side effects. These new drugs have been or will soon be in clinical trials to prove safety and efficacy as juvenile myositis therapies.

Cure JM-funded research is the reason for great optimism, as today we know that most JM kids will prevail in their battle with the disease and lead full, healthy, happy lives free from JM.

Executive Director Update - A Season of Gratitude

Season of Gratitude

I am writing to wish you and your family the very best this Holiday Season. This time of year, especially, I am filled with deep appreciation for all that Cure JM families have accomplished to advance our mission, to improve the lives of children living with JM, and to support the doctors and researchers driving better treatments as we fight for a cure.

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.

Join Cure JM

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Cure JM supports families, patients, and the juvenile myositis research community.

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