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CNSA-001
CNSA-001 is a small molecule that targets the underlying cause of a specific disease.
CNSA-001 is a small molecule that targets the underlying cause of a specific disease. Used for Spinal Muscular Atrophy.
At a glance
| Generic name | CNSA-001 |
|---|---|
| Also known as | sepiapterin |
| Sponsor | PTC Therapeutics |
| Modality | Small molecule |
| Therapeutic area | Rare Diseases |
| Phase | Phase 2 |
Mechanism of action
While the exact mechanism is not well understood, research suggests that CNSA-001 works by modulating a key biological pathway. This modulation leads to a cascade of effects that ultimately result in the desired therapeutic outcome.
Approved indications
- Spinal Muscular Atrophy
Common side effects
- Nausea
- Fatigue
- Headache
Key clinical trials
- A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia (PHASE1, PHASE2)
- A Study of CNSA-001 in Women With Diabetic Gastroparesis (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |