{'fraction_non_alphanumeric': 0.022153846153846152, 'fraction_numerical': 0.010461538461538461, 'mean_word_length': 5.474900398406374, 'pattern_counts': {'":': 0, '<': 0, '<?xml version=': 0, '>': 0, 'https://': 0, 'lorem ipsum': 0, 'www.': 0, 'xml': 0}, 'pii_count': 0, 'substrings_counts': 0, 'word_list_counts': {'cursed_substrings.json': 0, 'profanity_word_list.json': 0, 'sexual_word_list.json': 0, 'zh_pornsignals.json': 0}}
Spinal muscular atrophy care in the COVID‐19 pandemic era

Abstract The coronavirus disease 2019 (COVID‐19) pandemic has resulted in reorganization of healthcare settings affecting the delivery of clinical care to patients with spinal muscular atrophy (SMA). There is a concern that patients with SMA may be at increased risk of manifesting severe symptoms of COVID‐19. Currently approved therapies for SMA improve survival and motor function; however, their delivery requires an increased exposure to the health system and a dedicated healthcare team. In this study, we discuss consensus recommendations pertaining to care of SMA patients during the pandemic. We highlight that SMA treatments should not be perceived as elective. Decisions regarding the delay of treatments should be made with consideration of the potential risks of COVID‐19 exposure and the risk of that delay. We emphasize the importance of collaborative treatment decisions between the patient, family, and healthcare provider, considering any geographic‐ or institution‐specific policies and precautions for COVID‐19.


| INTRODUCTION
Coronavirus disease 2019  caused by the severe acute respiratory syndrome coronavirus-2 (SARS-CoV-2) is a pandemic and public health emergency. The virus spreads efficiently and rapidly from person to person, and COVID-19 symptoms include fever, cough, fatigue, shortness of breath, sore throat, headache, diarrhea, and reduced taste sensation. Severe manifestations, including pneumonia, acute respiratory distress syndrome, and death are more common in older patients and those with medical co-morbidities. [1][2][3] Nevertheless, several unexpected deaths have been reported in previously healthy young adults and teenagers. 4

,5 A recent US Centers for
Disease Control and Prevention mortality and morbidity weekly report note that the majority of hospitalized children with  in the United States for whom the information was available had one or more underlying medical conditions such as chronic lung disease, cardiovascular disease, and immunosuppression. 6 Management is supportive as there is no specific antiviral treatment currently available.
Social distancing, the most important intervention to limit the spread of COVID-19, 6-8 has necessitated reorganization of healthcare practice. Though emergent and urgent care continue to be provided to patients in hospital and office settings, elective and nonurgent services are now being provided by telehealth or are being rescheduled. 9 Currently available US Food and Drug Administration-approved therapies for spinal muscular atrophy (SMA), nusinersen and onasemnogene abeparvovec-xioi, [10][11][12] are life altering and are reshaping the natural history of the disease, resulting in improved survival and motor function. These treatments, however, are not a cure, and patients continue to live with substantial comorbidity, especially symptomatic infantile onset patients who may have pulmonary compromise and require daily supportive care. 13 Patients with SMA are at increased risk of respiratory infections in general, and may be at an increased risk of manifesting severe symptoms of COVID-19. Currently, there are no data on how the COVID-19 public health emergency and resultant healthcare restructuring have impacted the care of SMA patients. Both approved treatments require an increased exposure to the health system around their delivery. Peer-reviewed published literature pertaining to COVID-19 and SMA is lacking.
We assembled an expert panel of neuromuscular specialists to provide recommendations related to SMA care during this public health emergency.
Expert leaders in SMA from across the United States and Europe were identified and invited to join the panel. The primary mode of communication was email for thorough point-by-point review to reach a consensus. There were no dissenting opinions with regard to the final recommendations. This report, therefore, reflects the consensus opinion of the authors.
First and foremost, patients with SMA and their families should follow national, and local guidelines as well as any additional recommendations for people at risk for serious illnesses from COVID-19. 14,15 Patients and families should also follow the guidelines pertaining to COVID-19 at the institutions where they receive their health care.
Surging COVID-19 rates are placing a tremendous burden on the healthcare system and healthcare providers, resulting in interruption of elective and/or nonemergent services and procedures. In some instances, this has interfered with planned treatments for SMA patients. SMA treatments are critical to the health and wellbeing of these patients and should not be perceived as elective or nonurgent. This is especially true for young children with infantile onset SMA.
Nusinersen is given intrathecally with the recommended schedule of four loading doses in the first 2 months followed by maintenance doses every 4 months. The US package insert addresses missed nusinersen dosing, noting that if a loading dose is delayed or missed, administer it as soon as possible, with at least 14 days between doses, and continue dosing as prescribed. If a maintenance dose is delayed or missed, administer as soon as possible and continue dosing every 4 months. 10 In response to a request for more information from Biogen around this issue, the company was able to share pharmacokinetic SMA treatments require a team of dedicated healthcare personnel and a secure setting, which might be challenging during a pandemic. We strongly recommend that healthcare providers work collaboratively to avoid treatment delays. Early and uninterrupted treatment, particularly for children with infantile onset SMA, leads to better outcomes. 16,[18][19][20] We emphasize that treatment decisions should be individualized and made cohesively between the patient, family, and healthcare provider, taking into account any geographicor institution-specific policies and precautions for COVID-19.
Insurance providers typically require standardized physical therapy assessments before and on a regular basis following or during treatment to document the impact of such treatment. We believe that these assessments should appropriately be deferred to minimize exposure of these fragile patients; partial functional assessments by means of telemedicine may be feasible in some care contexts. We recommend that coverage for nusinersen and onasemnogene abeparvovec-xioi should be provided without interruption even if formal clinical and physical therapy assessments are limited. Patients' neurologists may discuss these needs with third-party payers. The clinical urgency of ongoing physical, occupational, and speech therapies should be evaluated on a case-by-case basis and their suspension or continuation agreed upon between providers and their patients.
Newborn screening (NBS) programs have allowed presymptomatic identification and treatment of SMA patients, dramatically improving outcomes. 21 NBS for SMA is currently practiced or piloted in more than 30 states in the United States. 22 The full impact of the COVID-19 pandemic on the SMA NBS programs is unclear.
However, this impact may include delay in implementing NBS programs in some states as healthcare resources are diverted; there may limited ability to arrange face to face patient evaluations and blood draws for testing while many medical practices severely limit in-person evaluations or transition to telehealth. 9 We continue to recommend urgent evaluation of infants with SMA identified by NBS with rapid initiation of treatment while following the regional and institutional policies pertaining to the public health emergency and maximizing the safety of patients and caregivers.
In summary, the COVID-19 pandemic presents tremendous challenges to the healthcare community, and disease-specific recommendations are rapidly evolving. We emphasize that:

ETHICAL PUBLICATION STATEMENT
We confirm that we have read the Journal's position on issues involved in ethical publication and affirm that this report is consistent with those guidelines.