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Spinal muscular atrophy (SMA)

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchHighest evidence only (55)Meta-analysis (4)Treatment guideline (3)Systematic review (20)Phase 3 study (16)Randomized controlled trial (12)Clinical trial (482)Review article (202)Early-stage study (phase 1 or 2) (13)Case report (127)Laboratory or animal study (39)Commentary or editorial (56) Studies currently recruiting participants only (34)

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Publications and studies (1659)

• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Thoracic Jia-Ji electro-acupuncture mitigates low skeletal muscle atrophy and improves motor function recovery following thoracic spinal cord injury in rats. (2022/11/15) ♡
• SMA-linked SMN mutants prevent phase separation properties and SMN interactions with FMRP family members. (2022/11/14) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Kennedy's disease presented with mastication fatigue combined with positive titin antibody: a case report. (2022/11/14) ♡
• PCR-Based Screening of Spinal Muscular Atrophy for Newborn Infants in Hyogo Prefecture, Japan. (2022/11/14) ♡
• Knowledge of genetic test results among caregivers and individuals with spinal muscular atrophy. (2022/11/08) ♡
• A link between agrin signalling and Ca(v)3.2 at the neuromuscular junction in spinal muscular atrophy. (2022/11/08) ♡
• p53-dependent c-Fos expression is a marker but not executor for motor neuron death in spinal muscular atrophy mouse models. (2022/11/07) ♡
• 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. Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology. (2022/11/05) ♡
• Thoracolumbar Sacral Orthosis for Spinal Fractures: What's the Evidence and Do Patients Use Them? (2022/11/05) ♡
• Robust Generation of Ready-to-Use Cryopreserved Motor Neurons from Human Pluripotent Stem Cells for Disease Modeling. (2022/11/03) ♡
• Clinical follow-up analysis of multidisciplinary treatment of children with spinal muscular atrophy. (2022/11/02) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Spinal Muscular Atrophy: Family and Provider Perspectives. (2022/11/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Anesthetic and surgical management of tracheotomy in a patient with Kennedy's Disease. (2022/11/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Expanding the spectrum of TBK1-related disorders: Adult-onset non-5q spinal muscular atrophy. (2022/11/01) ♡
• Assessment of health-related quality of life in patients with spinal muscular atrophy in China. (2022/11/01) ♡
• Analysis of scores of Symptom Checklist 90 (SCL-90) questionnaire of 182 parents of children with spinal muscular atrophy: a cross-sectional study. (2022/11/01) ♡
• Genetic and Clinical Spectrum of GNE Myopathy in Russia. (2022/10/31) ♡
• Population-based assessment of nusinersen efficacy in children with spinal muscular atrophy: a 3-year follow-up study. (2022/10/31) ♡
• Biological networks and complexity in early-onset motor neuron diseases. (2022/10/21) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A Novel Mutation in the NAGLU (N-Acetyl-Alpha-Glucosaminidase) Gene Associated With Mucopolysaccharidosis Type III-B in a Saudi Girl. (2022/10/20) ♡
• 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. Calpain-mediated proteolysis as driver and modulator of polyglutamine toxicity. (2022/10/19) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Celastrol and Rhynchophylline in the mitigation of simulated muscle atrophy under in vitro. (2022/10/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Switching therapies: safety profile of Onasemnogene abeparvovec-xioi in a SMA1 patient previously treated with Risdiplam. (2022/09/30) ♡
• Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets. (2022/09/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. X-linked myotubular myopathy associated with an MTM1 variant in a Maine coon cat. (2022/09/01) ♡
• Shenkang injection protects against renal fibrosis by reducing perforin expression through the STING/TBK1/IRF3 signaling pathways in natural killer cells. (2022/09/01) ♡
• 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. Update on spinal muscular atrophy treatment. (2022/08/30) ♡
• Association between serum zinc level and lipid profiles in children with spinal muscular atrophy. (2022/08/15) ♡
• Vitamin D inadequacy combined with high BMI affects paraspinal muscle atrophy and pain intensity in postmenopausal women. (2022/08/01) ♡
• Scoliosis in Spinal Muscular Atrophy Type 1 in the Nusinersen Era. (2022/08/01) ♡
• Coaxial Synthesis of PEI-Based Nanocarriers of Encapsulated RNA-Therapeutics to Specifically Target Muscle Cells. (2022/07/22) ♡
• 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) ♡

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