I welcome Agamree’s approval with renewed optimism

UK recently approved the treatment for patients 4 and older

by Shalom Lim | January 31, 2024

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I received an email on Jan. 12 from Duchenne UK, an important Duchenne muscular dystrophy (DMD) nonprofit in the United Kingdom. Its announcement: “Breaking News! First treatment for all [DMD] patients approved in U.K.”

It went on to note that the Medicines and Healthcare products Regulatory Agency, the U.K.’s version of the U.S. Food and Drug Administration (FDA), had approved the medication Agamree (vamorolone) for DMD patients 4 and older, regardless of their type of genetic mutation or ambulatory status. In October, the FDA had approved Agamree for DMD patients 2 and older in the U.S.

The power of hope

Duchenne UK explained that Agamree is an alternative to corticosteroids commonly used to manage DMD by reducing inflammation and preserving muscle strength. Unlike steroids, which have many adverse side effects that I’ve personally experienced, Agamree is a dissociative corticosteroid. That means it aims to offer DMD patients the benefits of corticosteroids while minimizing unwanted side effects, such as weight gain, the loss of bone density, stunted growth, and behavioral problems.

This news was encouraging, especially after I’d had a difficult start to 2024. It reminded me of the power of hope during hard times.

I’d learned about Agamree in early 2022 from a scientific adviser at CureDuchenne, a U.S.-based nonprofit that is similar to Duchenne UK. As a DMD patient who’s ineligible for most treatments because of my genetic mutation (I’m missing the first nine exons of the dystrophin gene), discovering that I can be treated with Agamree offered me fresh optimism for a longer life expectancy. That’s crucial, as grappling with the reduced life expectancy that comes with DMD has affected my mental health, causing me anxiety and other issues throughout my life.

It’s also heartening to discover that Agamree can treat people regardless of their mobility and motor function. I’ve been in a wheelchair for more than 20 years, and my breathing has declined to the extent that I rely on round-the-clock ventilator support to survive. My heart isn’t getting any stronger, either.

I hope that Agamree will be successful in treating me once it arrives here in Singapore. I think it’ll be a game changer for me and my fellow DMD survivors, which is why I still cling to hope for a better future.

Even so, Duchenne will always be a vital part of my identity as a disability advocate. My wish now is for Agamree to enable and empower me to advocate for the DMD community for years to come.

Note: Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy.

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About the Author

Shalom Lim Shalom Lim is a University of Liverpool graduate, having completed an honors bachelor’s degree in criminology and security in July 2021. Born with Duchenne muscular dystrophy and diagnosed at 4 months old in 1996, he is currently based in the Eastern part of Singapore. His hope for his column is to advocate for the health and well-being of the muscular dystrophy community in Southeast Asia.