Vamorolone FAQs in JM

Vamorolone FAQ

The FDA will soon be ruling on a new drug application for the use of vamorolone in Duchenne muscular dystrophy. We understand that families have questions surrounding vamorolone’s status for use in juvenile myositis. 

We have consulted with JM experts on the potential implications of a pending approval for the drug in another disease and what this currently means for JM patients.

Frequently Asked Questions & Answers

Question 1: What does a potential FDA approval for vamorolone in Duchenne muscular dystrophy mean for JM patients?

Answer: Currently, vamorolone needs to be further studied in JM, with safety and efficacy being primary considerations. Duchenne muscular dystrophy and juvenile dermatomyositis are different diseases and would require separate studies.

Question 2: Is vamorolone currently available to JM patients?

Answer: Currently, vamorolone is not a treatment option in JM. JDM is an autoimmune disease that causes significant inflammation.  We do not yet know if vamorolone would be effective and safe in treating JM and, if so, what dosage of vamorolone would be required.

Question 3: What are the needs or next steps for a similar trial approval in JM?

Answer: Cure JM is working with ReveraGen on the feasibility of a trial in JM.

Question 4: Why is prednisone a first line medication in treating JM?

Answer: Steroids are life-saving medications. Any other questions about steroids or other medications should be discussed with your doctor individually, as each situation and each patient is unique.  

Rare Disease Warriors

Since it is Rare Disease Month, we would like to take a moment and shine the spotlight on some of our amazing JM warriors.

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