Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy

Pfizer – Non Small Cell Lung Cancer With ALK or ROS1 Rearrangement Pfizer – Non Small Cell Lung Cancer Harboring Molecular Alterations Biogen – Infantile-onset Spinal Muscular Atrophy Novartis Pharmaceuticals – Non-small Cell Lung Cancer (NSCLC) Biogen – Infantile-onset Spinal Muscular Atrophy (SMA)

Personalized Medicine for SMA: a Translational Project Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL) Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study) Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA). Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular Atrophy STENTRODE™ WITH THOUGHT CONTROLLED DIGITAL SWITCH: An Early Feasibility Study (EFS) of the Safety of an Endovascular Motor Neuroprosthesis (MNP) Device in Participants With Severe Upper Limb Impairment. Schulze Muscular Dystrophy Ability Clinical Study Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam COMMAND Early Feasibility Study (EFS) Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy Long-term Follow-Up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials Hippotherapy in Children With Spinal Muscular Atrophy Newborn Screening for Spinal Muscular Atrophy (SMA) – a Proof of Principle Study Using Anonymised Blood Spots. Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration Pediatric Powered Wheelchair Standing Devices: An Exploratory Study Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulatory Children With Spinal Muscular Atrophy A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam Holter of Movement in Patients With SMA Undergoing Treatment. Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen Adults With SMA Treated With Nusinersen Metabolomics of Children With SMA Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor Effects of Power Mobility on Young Children With Severe Motor Impairments Optimum Insufflation Capacity in NMD Flu Vaccine Study in Neuromuscular Patients 2011 Reciprocating Gait Orthoses for Paraplegia Patients WREX Outcome Study National Registry for Egyptian Pediatric Neuromuscular Diseases Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions Phase II Study of Leuprolide and Testosterone for Men With Kennedy’s Disease or Other Motor Neuron Disease Establishing Novel Detection Techniques for Various Genetic-Related Diseases by Applying DHPLC Platform. A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants Multi-disease Carrier Screening Test Validation AveXis Managed Access Program Cohort for Access to AVXS-101 Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients Measuring Levels of SMN in Blood Samples of SMA Patients SMN Copy Number Distribution in Mali, West Africa Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System Sun May Arise on SMA : Newborn Screening of SMA in Belgium Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA® A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers Study of Feasibility to Reliably Measure Functional Abilities’ Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) Clinical Study of Spinal Muscular Atrophy Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA) Natural History of Types 2 and 3 SMA in Taiwan A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) 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) Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA) Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy A Trial of Hydroxyurea in Spinal Muscular Atrophy A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy Valproate and Levocarnitine in Children With Spinal Muscular Atrophy An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy European Registry of Patients With Infantile-onset Spinal Muscular Atrophy Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA) Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy Neonatal Spinal Muscular Atrophy (SMA) Screening Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy 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) A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) New Imaging Biomarkers for Muscular Diseases – Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy Mechanisms of Cell Death in Spinal Muscular Atrophy Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy Newborn Screening for Spinal Muscular Atrophy