Family News

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

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.

Read the Newsletter

Season of Gratitude

Affordable and Accessible Treatments for JM

The FDA’s Ruling on Vamorolone in DMD

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