Records containing: Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2023-03-14SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients
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CLINICAL_TRIALS study record update · 2023-03-14SMA Natural History Study Evaluating Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
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CLINICAL_TRIALS study record update · 2023-03-09Interlaminar Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy: a Retrospective Analysis of More Than 500 Consecutive Administrations
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CLINICAL_TRIALS study record update · 2023-03-08SMN Circular RNAs as Potential New Targets and Biomarkers for the Therapeutic Response in Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2023-02-16Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2022-12-23Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis
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CLINICAL_TRIALS study record update · 2022-12-05Schulze Muscular Dystrophy Ability Clinical Study
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CLINICAL_TRIALS study record update · 2022-11-30Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).
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CLINICAL_TRIALS study record update · 2022-11-17Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)
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CLINICAL_TRIALS study record update · 2022-11-10Metabolome of Children With Spinal Muscular Atrophy Treated With Nusinersen
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CLINICAL_TRIALS study record update · 2022-10-25Quantification of the Postural Muscles Mechanical Properties of Individuals With Spinal Muscular Atrophy Using Myotonometer: Intra- and Inter Examiner Reliability
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CLINICAL_TRIALS study record update · 2022-10-21Community's Participation of Children With Spinal Muscular Atrophy Using Power Mobility
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CLINICAL_TRIALS study record update · 2022-09-19Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old: a Quality-of-life Measure for Patients With Spinal Muscular Atrophy in Turkey
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CLINICAL_TRIALS study record update · 2022-08-17Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2022-02-14Newborn Screening for Spinal Muscular Atrophy (SMA) - a Proof of Principle Study Using Anonymised Blood Spots.
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CLINICAL_TRIALS study record update · 2022-01-26Antisense Oligonucleotide Therapy in Spinal Muscular Atrophy: An Observational Study in China
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CLINICAL_TRIALS study record update · 2022-01-11A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2
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CLINICAL_TRIALS study record update · 2021-11-10Public Awareness Level of SMA and DMD Muscle Disorders in Turkey, Attitudes Toward Screening of Newborn and Carrier and Physiotherapy Practice
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CLINICAL_TRIALS study record update · 2021-09-22Characterizing Longitudinal Outcomes in Adults With SMA Treated With Nusinersen
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CLINICAL_TRIALS study record update · 2021-09-13European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2021-06-29Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments
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CLINICAL_TRIALS study record update · 2021-04-27Characterization of the Clinical-epidemiological Profile of Patients With Spinal Muscular Atrophy (SMA) 5q Types II and III in Follow-up in the Brazilian Unified Public Health System: A Cross-sectional Observational Study (Registry)
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CLINICAL_TRIALS study record update · 2021-04-26Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women
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CLINICAL_TRIALS study record update · 2021-03-10Sun May Arise on SMA : Newborn Screening of SMA in Belgium
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CLINICAL_TRIALS study record update · 2021-02-21Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions