Kaur M, Bach JR. Mortality despite new generation medications for pediatric neuromuscular diseases and how to prevent it. World J Transl Med 2026; 12(3): 121646 [DOI: 10.5528/wjtm.121646]
Corresponding Author of This Article
Maninderpal Kaur, MD, Assistant Professor, Department of Medicine, Rutgers New Jersey Medical School, University Hospital, 150 Bergen Street, H245, Newark, NJ 07103, United States. mk1786@njms.rutgers.edu
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Respiratory System
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review-article
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Kaur M, Bach JR. Mortality despite new generation medications for pediatric neuromuscular diseases and how to prevent it. World J Transl Med 2026; 12(3): 121646 [DOI: 10.5528/wjtm.121646]
World J Transl Med. Sep 28, 2026; 12(3): 121646 Published online Sep 28, 2026. doi: 10.5528/wjtm.121646
Mortality despite new generation medications for pediatric neuromuscular diseases and how to prevent it
Maninderpal Kaur, John R Bach
Maninderpal Kaur, Department of Medicine, Rutgers New Jersey Medical School, University Hospital, Newark, NJ 07103, United States
John R Bach, Department of Physical Medicine and Rehabilitation, Rutgers New Jersey Medical School, Rutgers New Jersey Medical School, Newark, NJ 07103, United States
Co-first authors: Maninderpal Kaur and John R Bach.
Author contributions: Kaur M and Bach JR contributed equally to this work as co-first authors; Bach JR conceived and designed the research; Kaur M drafted the paper; Bach JR and Kaur M revised the paper for important intellectual content; both authors read and approved the final manuscript.
AI contribution statement: No AI was used for the manuscript.
Conflict-of-interest statement: All authors declare that they have no conflict of interest to disclose.
Corresponding author: Maninderpal Kaur, MD, Assistant Professor, Department of Medicine, Rutgers New Jersey Medical School, University Hospital, 150 Bergen Street, H245, Newark, NJ 07103, United States. mk1786@njms.rutgers.edu
Received: March 30, 2026 Revised: July 12, 2026 Accepted: July 28, 2026 Published online: September 28, 2026 Processing time: 158 Days and 4.3 Hours
Abstract
With severe respiratory muscle weakness of pediatric patients with progressive neuromuscular disorders, progressive hypercapnic ventilatory failure can result in cor pulmonale but, even more often, otherwise benign upper respiratory tract infections result in pneumonia, supplemental oxygen administration, hypercapnic respiratory failure, coma, intubation, failure to ventilator wean, then the options of tracheotomy or terminal extubation with O2 and morphine. While new medications for spinal muscular atrophy, Pompe disease, and Duchenne muscular dystrophy can slow deterioration, cough peak flows can become or remain ineffective to clear the airways to prevent those two options. This work reviewed publications that demonstrate that instead of weaning from ventilatory support to be extubated, it can be only by extubating or decannulating ventilator unweanable patients that permit them to wean back to any ventilator use regimens they used before hospitalization. Avoiding tracheostomies for mechanical ventilation can also result in over half a million dollars per year in cost savings as well as preserve quality of life.
Core Tip: New gene and RNA-based therapies slow progression of pediatric neuromuscular diseases but do not restore effective cough. When hypoxic episodes are treated with supplemental oxygen instead of noninvasive ventilatory support (NVS) and mechanical insufflation-exsufflation (MIE), CO2 narcosis, intubation, and unnecessary tracheostomy or death often follow. Extubating ventilator-unweanable patients directly to continuous NVS and MIE, rather than pursuing prolonged weaning trials, allows most to return to their prior ventilator-use regimens without tracheostomy. This approach also saves over $500000 per patient annually in nursing and medical costs while preserving quality of life.
