Table 3.
Current preclinical trials in Mecp2 deficient mice.
| Agent or Approach | Sponsor | Action |
|---|---|---|
| BDNF boosters | ||
| LM22A-4 (BDNF loop mimetic) | Sigma-Aldrich; RSRT | Partial agonist of TrkB receptors |
| Improves breathing | ||
| RP103 (cysteamine) | Raptor Pharmaceuticals |
Increases BDNF expression and release |
| Improves breathing | ||
| Serotonin agonists | ||
| Sarizotan | Newron | 5HT1 agonist |
| Pharmaceuticals | Improves breathing | |
| NLX-101 | Neurolixis | 5HT1 agonist |
| Improves breathing | ||
| NMDA receptors | ||
| GluN2A negative allosteric modulator | Mnemosyne Pharmaceuticals |
Improved visual cortical functioning |
| Ketamine | Approved drug | Non-competitive NMDA receptor antagonist |
| Improved respiratory and locomotor function | ||
| Antidepressants | ||
| REV-003 | Revive | Atypical antidepressant |
| (Tianeptine) | Therapeutics | Improves breathing |
| MECP2 gene | ||
| Read-through compounds | Research labs | Skip premature STOP codon in nonsense mutations |