ArticleNeurology. Clinical practice2025
Spinal Muscular Atrophy Update in Best Practices: Recommendations for Treatment Considerations.
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Article in Neurology. Clinical practice, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed.
- Trial
- Guidelines addressing Motor Neurone Disease (MND): a scoping review.Neurodegenerative disease management · 2026Article
- Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy.Muscle & nerve · 2026Review
- Gut microbiota and neuromuscular disorders: a comprehensive review.Arquivos de neuro-psiquiatria · 2026Review
- Pre-symptomatic treatment of spinal muscular atrophy: a strategic shift from concept to clinical practice.World journal of pediatrics : WJP · 2026Article
- Nusinersen rescues taurine deficiency in patients with type 1 Spinal Muscular Atrophy.Communications medicine · 2026Article
- Neuromuscular Mechanisms and Oxidative Stress in Skeletal Muscle Atrophy: Emerging Stem Cell and Gene-Based Therapeutic Strategies.Muscles (Basel, Switzerland) · 2026Review
- SMΝΔ7 mice show breathing and airflow defects with significant pathology of respiratory and oral tract tissues.Frontiers in cellular neuroscience · 2026Article
- Review
- The Integrated Approach in Patients with Spinal Muscular Atrophy in the Era of Early Diagnosis, Etiopathogenic Therapies and Multidisciplinary Standards of Care and Rehabilitation Interventions Leads to New Phenotypes.Life (Basel, Switzerland) · 2025Article
- Evaluation and Treatment of Thoracic Insufficiency Syndrome and Early-Onset Scoliosis.Journal of clinical medicine · 2025Article
- Five-year disease-modifying therapeutic experience of 102 Chinese paediatric 5q-spinal muscular atrophy: a retrospective analysis.Brain communications · 2025Article
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Authors and funding
12 authors.
Mary K SchrothCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0000-0002-1720-4849
Jennifer DeansCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0009-0008-7696-9199
Diana X Bharucha GoebelCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.
W Bryan BurnetteCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0000-0002-7637-5330
Basil T DarrasCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0000-0002-1536-8145
Bakri H ElsheikhCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0009-0002-4129-3498
Marcia V FelkerCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0000-0002-8287-024X
Andrea KleinCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0000-0003-2733-5911
Jena KruegerCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0009-0009-3471-3395
Crystal M ProudCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.
Aravindhan VeerapandiyanCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0000-0002-3065-3956
Robert J GrahamCure SMA (MKS); Clinical Care Education (JD), Cure SMA; Neurology and Pediatrics (DBG), Children's National, and National Institute of Neurological Diseases and Stroke, National Institutes of Health; Division of Neurology (WBB), Department of Pediatrics, Vanderbilt University; Department of Neurology (BTD), Boston Children's Hospital, Harvard Medical School; Department of Neurology (BHE), The Ohio State University Wexner Medical Center; Child Neurology (MVF), Indiana University; Division of Neuropediatrics (AK), Development and Rehabilitation, Department of Pediatrics, Inselspital, Bern University Hospital; Pediatric Neuromuscular (JK), Helen DeVos Children's Hospital; Neurology (CMP), Children's Hospital of the King's Daughters; Department of Pediatrics (AV), Division of Neurology, University of Arkansas for Medical Sciences, Arkansas Children's Hospital; and Department of Anesthesiology (RJG), Critical Care and Pain Medicine, Boston Children's Hospital, Harvard Medical School.ORCID https://orcid.org/0000-0002-8659-6313
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background and Objectives: Spinal muscular atrophy (SMA) is an autosomal recessive disorder caused by biallelic variants of the Methods: A systematic literature review was conducted on SMN-enhancing treatments for SMA and related studies. A working group of American and European health care providers with expertise in SMA care identified barriers and developed recommendations through a modified Delphi technique with serial surveys and feedback through virtual meetings to fill gaps for information where evidence is limited. A community working group of an individual living with SMA and caregivers provided insight and perspective on SMA treatments and support through a virtual meeting to guide recommendations. Results: The health care provider working group and the community working group agreed that when determining whether to start, change, add, or discontinue a treatment, essential considerations include patient and family/caregiver perspective, and treatment safety and side effects. When initiating treatment for patients newly diagnosed with SMA, important patient characteristics are age and Discussion: Sharing information about current knowledge of treatments and shared decision-making between health care providers and patients living with SMA and caregivers are essential to overcoming barriers to providing SMN-enhancing treatments.
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