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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Infarction, Muscular Disorders, Atrophic, Necrosis, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, TDP-43 proteinopathy, amyotrophic lateral sclerosis, brain disorder, brain infarction, brain ischemia, brain stem infarction, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, injury, ischemic disease, metabolic disease, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, nervous system injury, neurodegenerative disease, neuromuscular disease, palsy, progressive muscular atrophy, proteostasis deficiencies, quadriplegia, spinal cord disorder, spinal cord injury, spinal muscular atrophy, stroke disorder, vascular disorderRecruitingNot applicable2026-10-09NCT07521930
Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular DystrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, central nervous system disorder, hereditary disease, motor neuron disorder, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingNot applicable2026-10-09NCT07356063
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 22026-10-08NCT07287982
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 22026-10-08NCT03779334
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasms by Site, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Recurrence, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, male reproductive organ cancer, male reproductive system disorder, motor neuron disorder, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, prostate cancer, prostate disorder, spinal cord disorder, spinal muscular atrophyRecruitingPhase 32026-10-05NCT07723248
A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-10-02NCT07531719
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplamcentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 32026-10-02NCT07444476
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer DrugsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, male reproductive organ cancer, male reproductive system disorder, motor neuron disorder, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, prostate cancer, prostate disorder, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 1 / Phase 22026-10-02NCT07336446
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophy, spinal muscular atrophy, type IIIRecruitingPhase 22026-10-02NCT07047144
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 42026-10-02NCT05861986
A Registry of Cases of Spinal Muscular Atrophycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-09-30NCT07805278
A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate CancerCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Genital Diseases, Kennedy disease, Male Urogenital Diseases, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, male reproductive organ cancer, male reproductive system disorder, motor neuron disorder, neoplasm, nervous system disorder, neurodegenerative disease, neuromuscular disease, prostate cancer, prostate disorder, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-09-30NCT06764485
An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyNot yet recruitingPhase 32026-09-29NCT07846696
TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Kennedy disease, X-linked disease, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 1 / Phase 22026-09-29NCT07190300
An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersencentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyNot yet recruitingNot applicable2026-09-24NCT07838779
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Juvenile Spinal Muscular Atrophy, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proximal spinal muscular atrophy, spinal cord disorder, spinal muscular atrophyActive, not recruitingPhase 32026-09-24NCT04042025
A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi ArabiaDisease Attributes, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingNot stated2026-09-22NCT07737756
Long-Term Follow-up Study for Patients From AVXS-101-CL-101Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingNot stated2026-09-22NCT03421977
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 42026-09-18NCT07448610
A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patientscentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyNot yet recruitingNot stated2026-09-17NCT07767890
Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMAcentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyNot yet recruitingNot applicable2026-09-16NCT07822711
VRehab-SMA Phase 1.2central nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingNot applicable2026-09-15NCT07578454
GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular AtrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Muscular Atrophy, Spinal, Type II, Spinal Muscular Atrophies of Childhood, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyEnrolling by invitationNot applicable2026-09-11NCT06772402
Pediatric Spinal Muscular Atrophy (SMA) China Registrycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyActive, not recruitingNot stated2026-09-11NCT05042921
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapycentral nervous system disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophyRecruitingPhase 42026-09-10NCT05861999