Background The emergence of new treatments for spinal muscular atrophy (SMA) is revolutionary, especially for SMA type 1 (SMA1). Data on respiratory outcomes remain sparse and rely mostly on randomized clinical trials. We report our experience of Nusinersen‐treated SMA1 patients in real‐world settings. Methods Data from SMA1 patients treated with Nusinersen were prospectively collected between 1/2017 and 1/2020. Respiratory variables included the use of assisted ventilation, the use of mechanical insufflation‐exsufflation (MIE), respiratory complications, and death or treatment cessation due to respiratory reasons. Results Twenty SMA1 patients were assessed before and after 2 years of Nusinersen treatment which was initiated at a median age of 13.5 months (range, 1–184). At baseline, 16 patients were using assisted ventilation, eight noninvasive and eight invasive. Twelve patients were using permanent ventilation and four partial ventilation. After 2 years of treatment, there was no change in respiratory support among ventilated patients. All four patients who were free from respiratory support at baseline required the initiation of assisted ventilation during the study period. All 20 patients used MIE after 2 years of treatment. Two patients died from acute respiratory failure and one sustained severe brain injury. Four patients had chronic and/or recurrent atelectasis. Conclusion Most of our patients were stable in their need for assisted ventilation and did not worsen as expected in SMA1, nor did they improve as might be hoped. Future studies are needed to determine if earlier treatment with Nusinersen might result in respiratory outcomes superior to those reported in this real‐life study.

Objective: A national lockdown was implemented in Israel to slow the viral spread of COVID-19. We assessed the real-time effects of the lockdown on disease expression and lifestyle modifications in pediatric patients with chronic respiratory disorders.Methods: An anonymous electronic questionnaire was distributed during lockdown (March-May 2020) to caregivers of patients with chronic respiratory disorders. The primary outcome was change in disease expression and the secondary outcomes were changes in lifestyle and caregivers' emotional status. Results:The clinical status of one-third of the 445 participating patients (age 0-18 years) reportedly improved, including decreased respiratory symptoms (n = 133, 33%), exacerbation frequency (n = 147, 35%), and use of reliever medications (n = 101, 27.4%). The condition of~10% of the patients worsened. Clinical improvement was noted mostly in young patients <5 years (p = .001), asthmatic patients (p = .033), and patients with multiple underlying respiratory disorders (p = .015). Patients whose condition significantly worsened were more likely to be >5 years (p < .001), had increased screen time, decreased physical activity, and shorter sleep duration compared to their younger counterparts (p = .008, <.001, and .001, respectively). Caregivers' reports (n = 236 [58%]) of their own anxiety levels and perceptions of the patients' elevated health risk were increased, regardless of the children's actual clinical status.Conclusion: COVID-19 lockdown was associated with clinical improvement/stability for most of the surveyed children; however, their caregivers' anxiety level was heightened. An increased sedentary lifestyle was reported mostly in older children.

Objectives To investigate whether the three nationwide coronavirus disease 2019 (COVID‐19) lockdowns imposed in Israel during the full first pandemic year altered the traditional seasonality of pediatric respiratory healthcare utilization. Methods Month by month pediatric emergency department (ED) visits and hospitalizations for respiratory diagnoses during the first full COVID‐19 year were compared to those recorded for the six consecutive years preceding the pandemic. Data were collected from the patients' electronic files by utilizing a data extraction platform (MDClone © ). Results A significant decline of 40% in respiratory ED visits and 54%–73% in respiratory hospitalizations during the first COVID‐19 year compared with the pre‐COVID‐19 years were observed ( p < 0.001 and p < 0.001, respectively). The rate of respiratory ED visits out of the total monthly visits, mostly for asthma, peaked during June 2020, compared with proceeding years (109 [5.9%] versus 88 [3.9%] visits; p < 0.001). This peak occurred 2 weeks after the lifting of the first lockdown, resembling the “back‐to‐school asthma” phenomenon of September. Conclusions This study demonstrates important changes in the seasonality of pediatric respiratory illnesses during the first COVID‐19 year, including a new “back‐from‐lockdown” asthma peak. These dramatic changes along with the recent resurgence of respiratory diseases may indicate the beginnings of altered seasonality in pediatric pulmonary pathologies as collateral damage of the pandemic.