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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 (55) Studies currently recruiting participants only (34)

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Publications and studies (1657)

• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy (2025-03-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (2025-03-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Quality of Life and Participation of the Adult with Spinal Muscular Atrophy in France (2025-03-07) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy (2025-03-07) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA (2025-03-07) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA (2025-03-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases (2025-03-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy (2025-02-28) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of AC176 for the Treatment of Metastatic Castration Resistant Prostate Cancer (2025-02-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Holter of Movement in Patients with SMA Undergoing Treatment. (2025-02-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA). (2025-02-03) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) (2025-01-31) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma (2025-01-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Evaluation of Balance and Functionality in Postmenopausal Osteoporosis Using Quadriceps Femoris Muscle Thickness, Beyond Thoracic Hyperkyphosis (2025-01-08) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases (2025-01-03) ♡
• The VAPB Axis Precisely Coordinates the Timing of Motoneuron Dendritogenesis in Neural Map Development. (2024/12/31) ♡
• Risdiplam utilization, adherence, and associated health care costs for patients with spinal muscular atrophy: a United States retrospective claims database analysis. (2024/12/30) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Targeting STMN2 for neuroprotection and neuromuscular recovery in Spinal Muscular Atrophy: evidence from in vitro and in vivo SMA models. (2024/12/27) ♡
• Favorable response to ketogenic diet therapy in a patient with DYNC1H1-related epilepsy. (2024/12/26) ♡
• A cross-sectional and longitudinal evaluation of serum creatinine as a biomarker in spinal muscular atrophy. (2024/12/25) ♡
• Meta-analysisiAll studies on one question combined and calculated together. This is the strongest form of research that exists: a single loose study can be coincidence, dozens together much less so. The label says something about the design, not about the outcome — which can also be that something does NOT work. Functional Outcome Measures to Optimize Drug Development in Spinal and Bulbar Muscular Atrophy: Results From a Meta-Analysis of the Global SBMA Dataset. (2024/12/24) ♡
• Spinal muscular atrophy is also a disorder of spermatogenesis. (2024/12/20) ♡
• Effect of nusinersen on pulmonary function in children with spinal muscular atrophy in the plateau region: A pilot study. (2024/12/19) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Recombinant SMN protein synergizes with spinal muscular atrophy therapy to counteract pathological motor neuron phenotypes. (2024/12/17) ♡
• Development and validation of a one-step SMN assay for genetic testing in spinal muscular atrophy via MALDI-TOF MS. (2024/12/16) ♡
• 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 progressive myoclonic epilepsy: A case report from China with new ASAH1 variants. (2024/12/15) ♡
• 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. Cytoskeleton dysfunction of motor neuron in spinal muscular atrophy. (2024/12/12) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Treatment strategies for patients with spinal muscular atrophy. (2024/12/10) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Postural Tremor Caused by Hirayama Disease Mimicking Essential Tremor. (2024/12/10) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Insights into diagnostic difficulties in spinal muscular atrophy: a Case Report series. (2024/12/10) ♡
• Association between cardiometabolic diseases and the risk and progression of motor neuron diseases in Sweden: a population-based case-control study. (2024/12/10) ♡
• Usability and Safety of the ATLAS 2030 Robotic Gait Device in Children with Cerebral Palsy and Spinal Muscular Atrophy. (2024/12/10) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Hirayama's Disease: About a Clinical Observation. (2024/12/10) ♡
• Fiducial-marked laminectomy window for intrathecal medication administration after spinal fusion: illustrative case. (2024/12/09) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Longitudinal data collection in pediatric and adult patients with 5q spinal muscular atrophy in Latin America: LATAM RegistrAME study - a clinical registry study protocol. (2024/12/09) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Editorial: Mechanisms of neurodegeneration in amyotrophic lateral sclerosis and related disorders. (2024/12/05) ♡
• Spinal TNF-α receptor 1 is differentially required for phrenic long-term facilitation (pLTF) over the course of motor neuron death in adult rats. (2024/12/05) ♡
• The association between gait speed and falls in ambulatory adults with spinal muscular atrophy: a retrospective pilot study. (2024/12/04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study. (2024/12/03) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. The First Patient in Poland Treated for SMA with Nusinersen During Pregnancy. (2024/12/03) ♡
• Co-culture of postnatal mouse spinal cord and skeletal muscle explants as an experimental model of neuromuscular interactions. (2024/12/02) ♡
• Comprehensive copy number analysis of spinal muscular atrophy among the Iranian population. (2024/12/02) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. ASAH1 Variants Causing Spinal Muscular Atrophy Phenotype. (2024/12/01) ♡
• Complete genomic profiles of 1496 Taiwanese reveal curated medical insights. (2024/12/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Outcomes of early-treated infants with spinal muscular atrophy: A multicenter, retrospective cohort study. (2024/12/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Case Series Examining Clinic, Laboratory, and Physical Function After Administration of Nusinersen in Adults With Spinal Muscular Atrophy, a Single-Center Study. (2024/12/01) ♡
• Offering reproductive genetic carrier screening for cystic fibrosis, spinal muscular atrophy and fragile X syndrome: Views of Victorian general practitioners. (2024/12/01) ♡
• A self-reported Brazilian registry of 5q-spinal muscular atrophy: data on natural history, genetic characteristics, and multidisciplinary care. (2024/12/01) ♡
• Type I spinal muscular atrophy and disease modifying treatments: a nationwide study in children born since 2016. (2024/11/30) ♡
• 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. Neuromuscular diseases: genomics-driven advances. (2024/11/26) ♡

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