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Recent advances in upstream medical therapies for neuromuscular disorders suggest that the best outcomes result from their administration in the pre-symptomatic, and perhaps, neonatal period. Currently available therapies, and many other therapies in the pipeline soon to be considered by the Food and Drug Administration, suggest the importance of avoiding potential life-threatening disease complications. There is evidence that this is almost always possible with the use of respiratory muscle aids to avoid pneumonias and respiratory failure, but these are currently little understood and rarely offered by the medical community. However, restoring neuromuscular function necessitates keeping the patient alive and well.

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Publication history

Received: 22 January 2023
Accepted: 01 February 2023
Available online: 24 February 2023

Copyright

© 2023 The Author(s). Published by Elsevier Ltd on behalf of Tsinghua University Press.

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This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/).

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