Table 4.
Development stages of therapeutic strategies proposed for Wolfram syndrome 1.
| Chemical Chaperones | Phases of the Therapeutic Approaches |
|---|---|
| AMX0035 | Clinical phase |
| HTS (high-throughput screening) | Under study |
| ER calcium stabilizers | |
| NCS1/Ibudilast | Preclinical studies |
| Dantrolene | Phase Ib/IIa non-randomized, open-label, clinical trial (Clinical Trials.gov identifier: NCT028029268) |
| 2end Generation Calcium Stabilizer JTV-519 | Clinical phase |
| Drugs targeting ER stress | |
| Valproic acid | Phase III randomized, double-blind, placebo-controlled trial (Clinical Trials.gov identifier: NCT03717909). |
| GLP-1R agonists/liraglutide | Clinical trial off-label phase I |
| Gene Therapy | |
| AAV rescue by WT WFS1 | Preclinical studies |
| CRISPR/Cas9 mutant WFS1 gene editing |
Preclinical studies |
| Regenerative Medicine | |
| Cell-replacement therapy (iPSCs) |
Preclinical studies |
| Regenerative gene delivery (MANF) | Preclinical studies |