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valproate + levocarnitine
- Development stage
- Phase 3
- Lead developer
- AIIMS New Delhi
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Valproate + levocarnitine is a combination therapy investigated for the treatment of **Spinal Muscular Atrophy (SMA)**. Valproate (valproic acid) acts as a **histone deacetylase (HDAC) inhibitor**, which has been shown to increase the expression of the survival motor neuron (SMN) protein by promoting the inclusion of exon 7 in the SMN2 gene transcript. Because valproate treatment can lead to carnitine depletion—and SMA patients often have reduced carnitine synthesis due to low muscle mass—levocarnitine (L-carnitine) is co-administered to maintain metabolic function and mitigate potential valproate-induced hepatotoxicity or hypocarnitinemia. This combination was notably studied in a randomized, placebo-controlled trial conducted by the All India Institute of Medical Sciences (AIIMS).
- Brand names
- Valprol
- Other names
- valproic acid + levocarnitineVPA + L-carnitinevalproate sodium + levocarnitine
02
Targets
SLC22A5 (Organic cation/carnitine transporter 2)ABAT (4-aminobutyrate aminotransferase)SCN1A (Voltage-gated sodium channel protein type 1 subunit alpha)HDAC (HDAC family)
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