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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)

• Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. A Prospective, Crossover Survey Study of Child- and Proxy-Reported Quality of Life According to Spinal Muscular Atrophy Type and Medical Interventions. (2020/04/01) ♡
• Evaluation of body composition as a potential biomarker in spinal muscular atrophy. (2020/04/01) ♡
• Impact of scoliosis surgery on pulmonary function in patients with muscular dystrophies and spinal muscular atrophy. (2020/04/01) ♡
• Thirty years of translational research in Mobility Medicine: Collection of abstracts of the 2020 Padua Muscle Days. (2020/04/01) ♡
• Serum creatinine is a biomarker of progressive denervation in spinal muscular atrophy. (2020/03/03) ♡
• Preoperative factors that predict fair outcomes following surgery in patients with proximal cervical spondylotic amyotrophy. A retrospective study. (2020/03/01) ♡
• Psychological distress in partners of patients with amyotrophic lateral sclerosis and progressive muscular atrophy: what's the role of care demands and perceived control? (2020/03/01) ♡
• Loss of egli-1, the Caenorhabditis elegans Orthologue of a Downstream Target of SMN, Leads to Abnormalities in Sensorimotor Integration. (2020/03/01) ♡
• Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen. (2020/03/01) ♡
• 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. Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery. (2020/03/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. False Negative Carrier Screening in Spinal Muscular Atrophy. (2020/03/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Current and future treatments of spinal muscular atrophy. (2020/03/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A new case of SMABF2 diagnosed in stillbirth expands the prenatal presentation and mutational spectrum of ASCC1. (2020/03/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Chiari Malformation and Syringomyelia Associated with Hirayama Disease. (2020/03/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Intrathecal nusinersen treatment after ventriculo-peritoneal shunt placement: A case report focusing on the neurofilament light chain in cerebrospinal fluid. (2020/03/01) ♡
• Identification of novel SMN1 subtle mutations using an allelic-specific RT-PCR. (2020/03/01) ♡
• Trajectory of change in the swallowing status in spinal muscular atrophy type I. (2020/03/01) ♡
• Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' Reports. (2020/02/18) ♡
• AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy. (2020/02/05) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Enchanted voice. (2020/02/01) ♡
• Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Outcome measures in a cohort of ambulatory adults with spinal muscular atrophy. (2020/02/01) ♡
• Analysis of spinal muscular atrophy-like patients by targeted resequencing. (2020/02/01) ♡
• Nusinersen for older patients with spinal muscular atrophy: A real-world clinical setting experience. (2020/02/01) ♡
• ASC-1 Is a Cell Cycle Regulator Associated with Severe and Mild Forms of Myopathy. (2020/02/01) ♡
• 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. Transition to adult care in sleep medicine. (2020/02/01) ♡
• Long acting GLP-1 analog liraglutide ameliorates skeletal muscle atrophy in rodents. (2020/02/01) ♡
• 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. Expanding therapeutic opportunities for neurodegenerative diseases: A perspective on the important role of phenotypic screening. (2020/02/01) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Drosophila SMN2 minigene reporter model identifies moxifloxacin as a candidate therapy for SMA. (2020/02/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. FHL1-related myopathy may not be classified by reducing bodies in muscle biopsy. (2020/02/01) ♡
• Splicing Defects of the Profilin Gene Alter Actin Dynamics in an S. pombe SMN Mutant. (2020/01/24) ♡
• 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. The Role of RNA Binding Proteins for Local mRNA Translation: Implications in Neurological Disorders. (2020/01/15) ♡
• Diagnostic accuracy of MRI and ultrasound in chronic immune-mediated neuropathies. (2020/01/07) ♡
• Treatment guidelineiAn official agreement between doctors about how this disease should be treated. This is not a single study but the conclusion of an entire medical field. Gastrointestinal bleeding prophylaxis for critically ill patients: a clinical practice guideline. (2020/01/06) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Nusinersen Administration Via an Intrathecal Port in a 16-Year-Old Spinal Muscular Atrophy Patient with Profound Scoliosis. (2020/01/01) ♡
• 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. Spinal muscular atrophy with respiratory distress type 1: Clinical phenotypes, molecular pathogenesis and therapeutic insights. (2020/01/01) ♡
• Simple Fluoroscopy-Guided Transforaminal Lumbar Puncture: Safety and Effectiveness of a Coaxial Curved-Needle Technique in Patients with Spinal Muscular Atrophy and Complex Spines. (2020/01/01) ♡
• 244th ENMC international workshop: Newborn screening in spinal muscular atrophy May 10-12, 2019, Hoofdorp, The Netherlands. (2020/01/01) ♡
• 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 infantile spinal muscular atrophy (SMAX2) caused by novel c.1681G>A substitution in the UBA1 gene, expanding the phenotype. (2020/01/01) ♡
• 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. Alternative Splicing of ALS Genes: Misregulation and Potential Therapies. (2020/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. RESTORE: A Prospective Multinational Registry of Patients with Genetically Confirmed Spinal Muscular Atrophy - Rationale and Study Design. (2020/01/01) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen (2020-11-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy (2020-11-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (2020-10-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants (2020-10-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Flu Vaccine Study in Neuromuscular Patients 2011 (2020-10-19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA) (2020-10-05) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Prospective Evaluation of Infants With Spinal Muscular Atrophy: (2020-09-02) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study of CK-2127107 in Patients With Spinal Muscular Atrophy (2020-08-31) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. AveXis Managed Access Program Cohort for Access to AVXS-101 (2020-08-28) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Spinal Cord Gray Matter Imaging in Post Polio Syndrome (2020-06-24) ♡

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