Spinal Muscular Atrophy Type I
Rare Diseases
1
Pipeline Programs
2
Companies
3
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Leadiant BiosciencesMD - Rockville
1 program1
Valproic Acid and LevocarnitinePhase 1/21 trial
Active Trials
NovartisBASEL, Switzerland
2 programsIn-home body weight support harness systemN/A1 trial
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in SpainN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Leadiant BiosciencesValproic Acid and Levocarnitine
NovartisTherapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
NovartisIn-home body weight support harness system
Clinical Trials (3)
Total enrollment: 142 patients across 3 trials
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Start: Apr 2008Est. completion: Jun 201240 patients
Phase 1/2Completed
NCT06632730NovartisTherapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
Start: Jul 2023Est. completion: Nov 202369 patients
N/ACompleted
Body Weight Support Harness System in Spinal Muscular Atrophy
Start: Sep 2018Est. completion: Jul 202433 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 companies competing in this space