Therapeutic advances in spinal muscular atrophy
Author:
Publisher
Elsevier BV
Subject
Pediatrics, Perinatology and Child Health
Reference14 articles.
1. Risdiplam in spinal muscular atrophy type 1;Baranello;New Eng J Med,2021
2. Therapeutic strategies for spinal muscular atrophy: SMN and beyond;Bowerman;Dis Model Mech,2017
3. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the phase 2 NURTURE study;De Vivo;Neuromuscul Disord,2019
4. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study;Finkel;Lancet,2016
5. Diagnosis and management of spinal muscular atropy: Part 2: pulmonary and acute care; medications, supplements and immunisations; other organ systems and ethics;Finkel;Neuromuscul Disord,2018
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2. Diving into progress: a review on current therapeutic advancements in spinal muscular atrophy;Frontiers in Neurology;2024-05-24
3. The dynamic of changes of pNFH levels in the CSF compared with the motor scales’ scores during three years of nusinersen treatment in children with spinal muscular atrophy types 2 and 3;Balneo and PRM Research Journal;2024-03-31
4. Emerging antibody-based therapies for Huntington’s disease: current status and perspectives for future development;Expert Review of Neurotherapeutics;2024-02-07
5. New for 2022: Food and Drug Administration Approvals of Pediatric Respiratory Medications;Pediatric Allergy, Immunology, and Pulmonology;2023-05-09
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