{"disease":{"id":"spinal-muscular-atrophy-type-i","name":"spinal muscular atrophy type i"},"drugs":{"marketed":[{"drug_id":"pf-06954522","indication_name":"Spinal Muscular Atrophy (SMA) with a genetically confirmed diagnosis of SMA type 1, or SMA type 2 or 3 in patients with a body weight of 13 kg or less","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Pf-06954522","generic_name":"pf-06954522","company_name":"Pfizer","drug_phase":"marketed","molecular_target":"[specific target]","drug_class":"Small molecule inhibitor","quality_score":45,"revenue":null,"mechanism":""}],"pipeline":[],"offLabel":[],"totalMarketed":1,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT03655223","title":"Early Check: Expanded Screening in Newborns","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":30000,"lead_sponsor_name":"RTI International","has_results":false},{"nct_id":"NCT05626855","title":"Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":238,"lead_sponsor_name":"Scholar Rock, Inc.","has_results":false},{"nct_id":"NCT05156320","title":"Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":188,"lead_sponsor_name":"Scholar Rock, Inc.","has_results":false},{"nct_id":"NCT03709784","title":"Spinraza in Adult Spinal Muscular Atrophy","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":148,"lead_sponsor_name":"Washington University School of Medicine","has_results":false},{"nct_id":"NCT04042025","title":"Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi","phase":"PHASE3","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":85,"lead_sponsor_name":"Novartis Gene Therapies","has_results":false},{"nct_id":"NCT04907162","title":"Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders","phase":"","overall_status":"COMPLETED","enrollment_count":82,"lead_sponsor_name":"LMU Klinikum","has_results":false},{"nct_id":"NCT06632730","title":"Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain","phase":"","overall_status":"COMPLETED","enrollment_count":69,"lead_sponsor_name":"Novartis","has_results":false},{"nct_id":"NCT05839145","title":"Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation Study","phase":"NA","overall_status":"UNKNOWN","enrollment_count":60,"lead_sponsor_name":"Institut de Myologie, France","has_results":false},{"nct_id":"NCT03921528","title":"An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":58,"lead_sponsor_name":"Scholar Rock, Inc.","has_results":true},{"nct_id":"NCT06137612","title":"Spinal Cord Gray Matter Imaging in Spinal Muscular Atrophy","phase":"","overall_status":"UNKNOWN","enrollment_count":52,"lead_sponsor_name":"University Hospital, Basel, Switzerland","has_results":false},{"nct_id":"NCT07208903","title":"Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":36,"lead_sponsor_name":"University Hospital, Strasbourg, France","has_results":false},{"nct_id":"NCT05715749","title":"Body Weight Support Harness System in Spinal Muscular Atrophy","phase":"NA","overall_status":"COMPLETED","enrollment_count":33,"lead_sponsor_name":"Nationwide Children's Hospital","has_results":false},{"nct_id":"NCT06864767","title":"Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA","phase":"","overall_status":"COMPLETED","enrollment_count":23,"lead_sponsor_name":"Zeynep HOŞBAY","has_results":false},{"nct_id":"NCT07400198","title":"Gait and Bone Health in SMA","phase":"","overall_status":"RECRUITING","enrollment_count":22,"lead_sponsor_name":"Jacqueline Montes","has_results":false},{"nct_id":"NCT05878418","title":"The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy","phase":"NA","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"Medipol University","has_results":false},{"nct_id":"NCT05430113","title":"Spinal Cord Stimulation in Spinal Muscular Atrophy","phase":"NA","overall_status":"COMPLETED","enrollment_count":3,"lead_sponsor_name":"Marco Capogrosso","has_results":true},{"nct_id":"NCT05416034","title":"Exoskeleton Impact on the Quality of Life on Patients With Spinal Muscular Atrophy","phase":"NA","overall_status":"COMPLETED","enrollment_count":3,"lead_sponsor_name":"MarsiBionics","has_results":false},{"nct_id":"NCT03837184","title":"Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":2,"lead_sponsor_name":"Novartis Gene Therapies","has_results":true}],"total":18},"guidelines":[],"source":"Drug Landscape verified database"}