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Drug intelligence / Profile preview
SLX-100
- Development stage
- Unknown
- Lead developer
- Solaxa
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
SLX-100 is a uniquely formulated version of dalfampridine (also known as 4-aminopyridine), developed by Solaxa for the treatment of spinocerebellar ataxia (SCA), specifically targeting spinocerebellar ataxia type 27B (SCA27B). Dalfampridine is an aminopyridine compound that acts as a broad-spectrum potassium channel blocker. By inhibiting voltage-gated potassium channels in neurons, it prolongs action potentials and enhances synaptic transmission in demyelinated or dysfunctional nerve fibers. This mechanism aims to restore nerve function and improve motor coordination in patients with SCA. SLX-100 has received orphan drug designation from the FDA and is currently being evaluated in registrational Phase 3 clinical trials for SCA27B[1][3][6][8].
- Other names
- repurposed 4-Aminopyridinerepurposed4-Aminopyridinerepurposed-4-Aminopyridinedalfampridine
02
Targets
Kir (Inward-rectifier potassium channels)KCNQ1 (Potassium voltage-gated channel subfamily KQT member 1)
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