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Spinal muscular atrophy (SMA)
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Publications (1202)
- Clinical follow-up analysis of nusinersen in the disease-modifying treatment of pediatric spinal muscular atrophy. (2022/07/02) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Supercharged End-to-Side Anterior Interosseous to Ulnar Motor Nerve Transfer for Hirayama Disease: A Case Report. (2022/07/01) ♡
- Nutrition status survey of type 2 and 3 spinal muscular atrophy in Chinese population. (2022/07/01) ♡
- Evolution of bulbar function in spinal muscular atrophy type 1 treated with nusinersen. (2022/07/01) ♡
- Survival motor neuron protein deficiency alters microglia reactivity. (2022/07/01) ♡
- Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data. (2022/07/01) ♡
- Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. (2022/07/01) ♡
- Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. (2022/07/01) ♡
- Physical Therapy and Nusinersen Impact on Spinal Muscular Atrophy Rehabilitative Outcome. (2022/06/06) ♡
- Family, healthcare professional, and societal preferences for the treatment of infantile spinal muscular atrophy: A discrete choice experiment. (2022/06/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Reply to: The 4-Copy Conundrum in the Treatment of Infants with Spinal Muscular Atrophy. (2022/06/01) ♡
- Real-World Data on Access to Standards of Care for People With Spinal Muscular Atrophy in the UK. (2022/05/30) ♡
- Overview of Neuromuscular Disorder Molecular Diagnostic Experience for the Population of Latvia. (2022/05/16) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. The Advent of Omics Sciences in Clinical Trials of Motor Neuron Diseases. (2022/05/07) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Challenging times: Delivering gene therapies and an opportunity for shared learning. (2022/05/01) ♡
- Integrating newborn screening for spinal muscular atrophy into health care systems: an Australian pilot programme. (2022/05/01) ♡
- Body mass index in type 2 spinal muscular atrophy: a longitudinal study. (2022/05/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Management of Spinal Muscular Atrophy in the Adult Population. (2022/05/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of real-life outcome data of patients with spinal muscular atrophy treated with nusinersen in Switzerland. (2022/05/01) ♡
- Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. Systematic Literature Review to Assess the Cost and Resource Use Associated with Spinal Muscular Atrophy Management. (2022/04/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Late-onset idiopathic focal dystonia of the trunk: A treatable cause of bent spine syndrome. (2022/04/01) ♡
- Patient and Caregiver Treatment Preferences in Type 2 and Non-ambulatory Type 3 Spinal Muscular Atrophy: A Discrete Choice Experiment Survey in Five European Countries. (2022/04/01) ♡
- Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. Systematic Literature Review to Identify Utility Values in Patients with Spinal Muscular Atrophy (SMA) and Their Caregivers. (2022/04/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Dramatic Innovations in the Treatment of Spinal Muscular Atrophy, But Many Unknowns Remain. (2022/04/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Spinal muscular atrophy with predominant lower extremity (SMA-LED) with no signs other than pure motor symptoms at the intersection of multiple overlap syndrome. (2022/04/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Genetic architecture of motor neuron diseases. (2022/03/15) ♡
- Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model. (2022/03/02) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. An infant with congenital respiratory insufficiency and diaphragmatic paralysis: A novel BICD2 phenotype? (2022/03/01) ♡
- Anaesthetic considerations in posterior instrumentation of scoliosis due to spinal muscular atrophy: Case series of 56 operated patients. (2022/03/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Splicing efficiency of minor introns in a mouse model of SMA predominantly depends on their branchpoint sequence and can involve the contribution of major spliceosome components. (2022/03/01) ♡
- Comparative All-Cause Mortality Among a Large Population of Patients with Spinal Muscular Atrophy Versus Matched Controls. (2022/03/01) ♡
- Curcumin and neurological diseases. (2022/03/01) ♡
- Ethical Perspectives on Treatment Options with Spinal Muscular Atrophy Patients. (2022/03/01) ♡
- Identification of specific gene methylation patterns during motor neuron differentiation from spinal muscular atrophy patient-derived iPSC. (2022/02/15) ♡
- Structurally Mapping Antigenic Epitopes of Adeno-associated Virus 9: Development of Antibody Escape Variants. (2022/02/09) ♡
- Access to Innovative Neurological Drugs in Europe: Alignment of Health Technology Assessments Among Three European Countries. (2022/02/04) ♡
- Intramuscular tetanus neurotoxin reverses muscle atrophy: a randomized controlled trial in dogs with spinal cord injury. (2022/02/01) ♡
- Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. The predictive value of preoperative paraspinal muscle morphometry on complications after lumbar surgery: a systematic review. (2022/02/01) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Peripherally delivered Adeno-associated viral vectors for spinal cord injury repair. (2022/02/01) ♡
- Muscle "islands": An MRI signature distinguishing neurogenic from myopathic causes of early onset distal weakness. (2022/02/01) ♡
- Natural history of 10-meter walk/run test performance in spinal muscular atrophy: A longitudinal analysis. (2022/02/01) ♡
- Newborn screening for spinal muscular atrophy: The Wisconsin first year experience. (2022/02/01) ♡
- Assessment of Health-Related Quality of Life in Adult Spinal Muscular Atrophy Under Nusinersen Treatment—A Pilot Study. (2022/01/24) ♡
- Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries. (2022/01/21) ♡
- A Compound Heterozygous Mutation in Calpain 1 Identifies a New Genetic Cause for Spinal Muscular Atrophy Type 4 (SMA4). (2022/01/19) ♡
- A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data. (2022/01/17) ♡
- Development of the SMA Independence Scale—Upper Limb Module (SMAIS-ULM): A novel scale for individuals with Type 2 and non-ambulant Type 3 SMA. (2022/01/15) ♡
- Parent Perceptions in Choosing Treatment for Infants With Spinal Muscular Atrophy Diagnosed Through Newborn Screening. (2022/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study. (2022/01/01) ♡
- Axonal excitability changes in children with spinal muscular atrophy treated with nusinersen. (2022/01/01) ♡
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