Abatacept Trial Concludes With Favorable Results In JM Treatment

Abatacept Trial concludes with favorable results in JM treatment

A clinical trial treating patients with juvenile myositis with the drug abatacept resulted in lower disease activity and clinically significant responses in most patients. The trial was conducted at the Cure JM Center of Excellence at George Washington University, and preliminary results were presented at the Global Conference on Myositis.

Abatacept was generally well tolerated with minimal side effects, and the preliminary data suggest that abatacept may be beneficial in the treatment of hard-to-treat JM patients. Importantly, patients using abatacept improved at the same time prednisone dosage was reduced.  

The ten JM patients enrolled in the pilot study were between the ages of seven and 17, and all had moderate disease activity and moderately decreased functional disability. Five of the ten patients met the definition of improvement at week 12 of the trial, and nine met the definition by week 24. At week 24, three of the ten patients saw major improvement, four saw moderate improvement, and two saw minimal improvement when using Core Set Measures against a scale utilized by the International Myositis Assessment and Clinical Studies Group (IMACS).  

When assessing improvements in Core Set Measures against a similar scale used by the Pediatric Rheumatology International Trials Organization (PRINTO), seven of the nine improved patients saw major improvements in their scores. One patient was discontinued from the trial due to worsening interstitial lung disease.

“These results are preliminary but are certainly very encouraging,” said Dr. Rodolfo Curiel, the trial’s principal investigator. 

“We have much more data to assess before we publish the results, yet the pilot study showed that patients with refractory JDM treated with abatacept resulted in lower disease activity and clinically significant responses in the majority of patients.” Dr. Curiel is the Director of the Cure JM Center of Excellence at George Washington University.

Sydney’s Story

Spring break should be about fun and relaxation. Sandi and her husband Zack expected their trip to Mexico would be the same. Of course, taking

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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