[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"spinal-muscular-atrophy-1\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:spinal-muscular-atrophy-1":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,44],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100635610","phase-1-a-phase-iii-clinical-study-to-evaluate-skg0201-injection-in-subjects-with-spinal-muscular-atrophy-type-i-100635610",false,"NCT07554924","A Phase I\u002FII Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I","A Multicenter, Open-label, Dose-escalation Phase I\u002FII Clinical Study to Evaluate the Safety, Preliminary Efficacy and Immunogenicity of SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I","Inclusion Criteria:\n\n1. Type I SMA, defined by bi-allelic mutations in the SMN1 gene.\n2. Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control.\n3. On the day of administration, the age of the subjects do not exceed 180 days after birth.\n\nExclusion Criteria:\n\n1. Pulse oximetry \\\u003C 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.\n2. Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards.\n3. Active viral infection.\n4. In the presence of other severe infections or diseases that require systemic anti-infection treatment.\n5. Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients.\n6. Clinically significant abnormal laboratory values prior to administration.\n7. Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs.\n8. Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.","ALL","180 Days",{"count":19,"type":20},11,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","This is a phase I\u002FII clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.",[27],"Spinal Muscular Atrophy 1",[29,30],"SMA type I","SMA I","NOT_YET_RECRUITING","2026-06-04",{"date":34,"type":35},"2026-06-08","ACTUAL",{"date":37,"type":20},"2026-05-15",{"date":39,"type":20},"2029-05-25",{"name":41,"class":42},"Lanyue Biotech (Hangzhou) Co., Ltd.","INDUSTRY",3,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":16,"minAge":51,"maxAge":4,"enrollmentInfo":52,"targetDuration":4,"studyType":54,"phases":4,"briefSummary":55,"conditions":56,"keywords":57,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":74},"100640063","exploring-lived-experiences-of-families-of-children-with-spinal-muscular-atrophysma-type-1-regarding-feeding-and-communication-100640063","NCT07596277","Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication","Lived Experiences of Families of Children With Spinal Muscular Atrophy Type 1: A Qualitative Investigation Into Feeding and Communication","Inclusion Criteria:\n\n* Parents\u002Fguardians of children with a diagnosis of SMA1 who have received any one or more disease modifying therapies\\[CE7.1\\]\\[BA7.2\\]\\[AB7.3\\]\n* Participants need to be able to carry out interview in English In addition to parents, grandparents or other relatives with full parental responsibility will be included\n\nExclusion Criteria:\n\n* Parents\u002Fcarers who require an interpreter will not be included within the study for reasons of time and cost and because parents may feel less able to be open and honest when communicating with the researcher through a third party.\n* Primary carer who is a foster carer or corporate parent (i.e. a looked after child) as they are not likely to have the same decision-making 'freedoms'.","16 Years",{"count":53,"type":20},15,"OBSERVATIONAL","Spinal Muscular Atrophy Type 1 (SMA )Type 1 is a severe, early-onset neuromuscular condition that typically leads to profound weakness and impaired bulbar function-affecting swallowing, feeding, speech, and airway protection. Historically, bulbar decline contributed significantly to early morbidity and mortality.\n\nThe advent of disease-modifying therapies (DMTs) such as nusinersen, zolgensma and risdiplam (also known as Spinraza, Zolgensma, and Evrysdi) sinersinhas altered the clinical course of SMA Type 1, with emerging evidence of motor improvement and increased survival. However, the impact of these therapies on bulbar function remains poorly understood, and standardised tools for its assessment are lacking.\n\nQualitative research which uses interviews with parents and carers offers an opportunity to capture nuanced caregiver perspectives, identify meaningful functional outcomes, and explore daily lived experiences in a way quantitative tools currently cannot.\n\nThis study will investigate the lived experiences of families managing feeding and communication in children with SMA Type 1.\n\nThe research will also aim to\n\n1 Identify emotional, social issues experienced by families and practical support needs related to feeding and communication.\n\n2\\. Provide insights that can inform healthcare interventions and support",[27],[27,58,59,60,61,62,63],"Bulbar function","eating and drinking","dysphagia","Communication","speech","language","2026-05-29",{"date":66,"type":35},"2026-06-02",{"date":68,"type":20},"2026-06-19",{"date":70,"type":20},"2026-11-01",{"name":72,"class":73},"Guy's and St Thomas' NHS Foundation Trust","OTHER",1]