The effects of palovarotene in patients with fibrodysplasia ossificans progressiva: a plain language summary

What is this summary about? This is a plain language summary of an article originally published in the Journal of Bone and Mineral Research. People with fibrodysplasia ossificans progressiva (FOP) become physically disabled over time as new bone forms in places where it is not usually found, such as...

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Bibliographic Details
Published in:Future Rare Diseases 2023, Vol.3 (1)
Main Authors: Pignolo, Robert J, Hsiao, Edward C, Mukaddam, Mona Al, Baujat, Geneviève, Berglund, Staffan K, Brown, Matthew A, Cheung, Angela M, Cunto, Carmen De, Delai, Patricia, Haga, Nobuhiko, Kannu, Peter, Keen, Richard, Quan Sang, Kim-Hanh Le, Mancilla, Edna E, Marino, Rose, Strahs, Andrew, Kaplan, Frederick S
Format: Article
Language:English
Subjects:
fibrodysplasia ossificans progressive
palovarotene
plain language summary
rare disease
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Summary:What is this summary about? This is a plain language summary of an article originally published in the Journal of Bone and Mineral Research. People with fibrodysplasia ossificans progressiva (FOP) become physically disabled over time as new bone forms in places where it is not usually found, such as in muscles and ligaments. Until recently, there were no treatments for FOP that had been proven through clinical trials; however, a drug called palovarotene has been tested in clinical trials and may be effective. Here, we describe the MOVE trial, which investigated how effectively palovarotene works, as well as its safety in treating patients with FOP. What were the results? Results from MOVE suggest that palovarotene may reduce extra bone formation outside the normal skeleton. Patients with FOP who took palovarotene formed less new bone than those who did not take palovarotene. The most common side effects involved the skin, and included dryness and irritation. Some children who were still growing when they took palovarotene had a side effect that resulted in the (normal) growth of their skeleton stopping too soon. What do the results of the trial mean? Palovarotene may be a useful treatment option for FOP. Patients, caregivers, and doctors would need to consider the benefits and risks of treatment with palovarotene, particularly with growing children.
ISSN:2399-5262
2399-5270
2399-5270
DOI:10.2217/frd-2022-0015