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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)

• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Combined Exercise and Nutrition Intervention for Spinal Sarcopenia (2021-03-22) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium (2021-03-10) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function (2021-02-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions (2021-02-21) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) (2021-02-18) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) (2021-02-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (2021-02-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy (2021-02-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). (2021-02-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) (2021-02-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) (2021-02-16) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) (2021-02-16) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. AR, IGF-IR, IR and Peripheral Artery Disease (2021-01-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy (2021-01-05) ♡
• Continuous lengthening potential after four years of magnetically controlled spinal deformity correction in children with spinal muscular atrophy. (2020/12/30) ♡
• 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. Circulating microRNAs as potential biomarkers and therapeutic targets in spinal muscular atrophy. (2020/12/25) ♡
• 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. How to Build and to Protect the Neuromuscular Junction: The Role of the Glial Cell Line-Derived Neurotrophic Factor. (2020/12/24) ♡
• Cognitive Performance of Patients with Adult 5q-Spinal Muscular Atrophy and with Amyotrophic Lateral Sclerosis. (2020/12/23) ♡
• Development and Use of Gene Therapy Orphan Drugs-Ethical Needs for a Broader Cooperation Between the Pharmaceutical Industry and Society. (2020/12/23) ♡
• Mitochondrial defects in the respiratory complex I contribute to impaired translational initiation via ROS and energy homeostasis in SMA motor neurons. (2020/12/22) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Rare cause of unilateral upper limb weakness in a young adolescent. (2020/12/21) ♡
• Gene Therapy for Monogenic Inherited Disorders. (2020/12/21) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Exosc2 deficiency leads to developmental disorders by causing a nucleotide pool imbalance in zebrafish. (2020/12/17) ♡
• 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. Metabolic and Nutritional Issues Associated with Spinal Muscular Atrophy. (2020/12/16) ♡
• 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. Current Genetic Survey and Potential Gene-Targeting Therapeutics for Neuromuscular Diseases. (2020/12/16) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Spinal Muscular Atrophy Type 1 With Exon 8 Deletion and Bilateral Optic Atrophy. (2020/12/15) ♡
• 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. Advances in Newborn Screening and Presymptomatic Diagnosis of Spinal Muscular Atrophy. (2020/12/15) ♡
• Aberrant interaction of FUS with the U1 snRNA provides a molecular mechanism of FUS induced amyotrophic lateral sclerosis. (2020/12/11) ♡
• U2AF65-Dependent SF3B1 Function in SMN Alternative Splicing. (2020/12/09) ♡
• 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 Neuromuscular Junction in Health and Disease: Molecular Mechanisms Governing Synaptic Formation and Homeostasis. (2020/12/03) ♡
• The Burden of Spinal Muscular Atrophy on Informal Caregivers. (2020/12/02) ♡
• First Oral Drug Approved for Spinal Muscular Atrophy. (2020/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Sleep and Breathing After Nusinersen Therapy in a Child With Spinal Muscular Atrophy. (2020/12/01) ♡
• 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 Exaggerated Deep Tendon Reflexes and Scapular Winging: An Atypical Presentation. (2020/12/01) ♡
• Impact of a national population-based carrier-screening program on spinal muscular atrophy births. (2020/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Letter to the Editor Regarding "Chiari Malformation and Syringomyelia Associated with Hirayama Disease". (2020/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. In Reply to the Letter to the Editor Regarding "Chiari Formation and Syringomyelia Associated with Hirayama Disease". (2020/12/01) ♡
• Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Long-term follow-up of patients with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA) treated with olesoxime in the OLEOS trial. (2020/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Possible implication of undescribed SMN1-SMN2 genotype in chronic EMG-pattern of SMA with transitory acute denervation. (2020/12/01) ♡
• [Hirayama Disease can be Caused by Loss of Attachment of the Cervical Posterior Dura to the Pedicle due to Immunological Abnormalities of the Dura and Posterior Ligaments: A New Hypothesis]. (2020/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Infantile spinal muscular atrophy (SMA). (2020/12/01) ♡
• 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 (SMA) type I (Werdnig-Hoffmann disease). (2020/12/01) ♡
• 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. Clinical features of spinal muscular atrophy (SMA) type 2. (2020/12/01) ♡
• 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. Clinical features of spinal muscular atrophy (SMA) type 3 (Kugelberg-Welander disease). (2020/12/01) ♡
• 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. Respiratory management of children with spinal muscular atrophy (SMA). (2020/12/01) ♡
• 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. Pathogenesis and therapeutic targets in spinal muscular atrophy (SMA). (2020/12/01) ♡
• 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. Functional and surgical treatments in patients with spinal muscular atrophy (SMA). (2020/12/01) ♡
• 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. State of the art for motor function assessment tools in spinal muscular atrophy (SMA). (2020/12/01) ♡
• 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. Multidisciplinary approach and psychosocial management of spinal muscular atrophy (SMA). (2020/12/01) ♡
• 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. Ethical aspects in the care of a child with infantile spinal muscular atrophy (SMA). (2020/12/01) ♡

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