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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 extremely expensive therapies in the pipeline soon to be considered by the Food and Drug Administration, and suggest the importance of avoiding potential life-threatening disease complications for patients to continue to benefit from these. 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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What advances in upstream medical therapies inform neurorestoratology

Show Author's information John R. Bach( )
Department of Physical Medicine and Rehabilitation, Rutgers University - New Jersey Medical School, Center for Ventilator Management Alternatives, Pulmonary Rehabilitation of the University Hospital of Newark, Newark, NJ, 07102, USA

Abstract

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 extremely expensive therapies in the pipeline soon to be considered by the Food and Drug Administration, and suggest the importance of avoiding potential life-threatening disease complications for patients to continue to benefit from these. 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.

Keywords: Neurorestoratology, Gene therapy, Neuromuscular disease, Noninvasive ventilation

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Publication history

Received: 22 January 2023
Accepted: 02 February 2023
Published: 24 February 2023
Issue date: June 2023

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© 2023 The Author.

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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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