Spinal Muscular Atrophy Type 3
Rare Diseases
1
Pipeline Programs
3
Companies
3
Clinical Trials
1 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
3 companies ranked by most advanced pipeline stage
Catalyst PharmaceuticalsCORAL GABLES, FL
1 program1
Amifampridine Phosphate 10 MG Oral TabletPhase 21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Catalyst PharmaceuticalsAmifampridine Phosphate 10 MG Oral Tablet
BiogenGait and Bone Health in SMA
RocheSpinal Cord Stimulator
Clinical Trials (3)
Total enrollment: 25 patients across 3 trials
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
Start: Mar 2019Est. completion: Sep 2021
Phase 2Terminated
Gait and Bone Health in SMA
Start: Feb 2026Est. completion: Jun 202722 patients
N/ARecruiting
Spinal Cord Stimulation in Spinal Muscular Atrophy
Start: Apr 2022Est. completion: Aug 20273 patients
N/AActive Not Recruiting
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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
1 actively recruiting trials targeting 25 patients
3 companies competing in this space