Spinal Muscular Atrophy (SMA)
Rare Diseases
1
Pipeline Programs
3
Companies
3
Clinical Trials
2 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2022
2023
2024
2025
2026
2027
2028
2029
argenxARGX-119 IV
BiogenRisdiplam
BiogenVirtual reality
Clinical Trials (3)
Total enrollment: 723 patients across 3 trials
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 Atrophy
Start: Dec 2025Est. completion: May 202960 patients
Phase 2Recruiting
Adult SMA Research and Clinical Hub
Start: Jul 2022Est. completion: Apr 2027600 patients
N/ARecruiting
Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy
Start: May 2022Est. completion: Jul 202463 patients
N/ACompleted
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Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
2 actively recruiting trials targeting 723 patients
3 companies competing in this space