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. 2021 Oct 23;16:446. doi: 10.1186/s13023-021-02048-0

Table 3.

Short-listed repurposed treatments for SSMD and GPX4-related diseases

Name Rationale Availability SSMD Status
Vitamin E Potent antioxidant known to prevent ferroptosis Over-the-counter

Administered to 2 of 4 patients

Dosage: 15 mg 2X/day

N-Acetyl-Cysteine (NAC) Increases glutathione biosynthesis to boost residual GPX4 activity Over-the-counter

Administered to 2 of 4 patients

Dosage: 300 mg 3X/day

CoQ10 Essential for repair of peroxidized lipids. Acts as an antioxidant Over-the-counter

Administered to 2 of 4 patients

Dosage: 50 mg 2X/day

Selenium Limiting step in GPX4 production, may increase selenoprotein expression Over-the-counter

Administered to 1 of 4 patients

Dosage: 75 µg 1X/day

L-methionine Increases glutathione biosynthesis through transsulferation pathway to boost residual GPX4 activity Over-the-counter Pending administration
RT001 Protects lipid membranes against peroxidation In clinical trials for treating multiple indications Administered to 2 of 4 patients under Expanded Access
Dimethyl fumarate Activates Nrf2 gene, master regulator oxidative stress response Approved in USA for treating multiple sclerosis Evaluating for of off-label use

Collection of potential and in-use repurposed treatments for current SSMD patients