Table 2.
Stabilization of transthyretin tetramer mutants by tafamidis in an ex vivo study [105]
| Mutation | Measurable vehicle (DMSO) FOI values | Stabilization demonstrateda |
|---|---|---|
| Cys10Arg | Yes | Yes |
| Asp18Glu | Nob; Retest: Yes | Yesc; Retest: Yes |
| Pro24Ser | Yes | Equivocald |
| Ala25Ser | Yes | Yes |
| Val30Gly | Nob; Retest: Yes | Yesc; Retest: Yes |
| Arg34Ser | Yes | Yes |
| Arg34Thr | Yes | Yes |
| Lys35Thr | Yes | Yes |
| Ala36Pro | Yes | Yes |
| Asp38Ala | Yes | Yes |
| Trp41Leu | Yes | Yes |
| Gly47Glu | Yes | Yes |
| Thr49Ala | Yes | Yes |
| Ser50Arg | Yes | Yes |
| Glu54Gln | Yes | Yes |
| Glu54Lys | Yes | Yes |
| Leu55Gln | Yes | Yes |
| Thr56Lys | Yes | Yes |
| Phe64Ser | Nob | Yesc |
| Tyr69His | Yes | Yes |
| Val71Ala | Yes | Yes |
| Tyr78Phe | Nob | Yesc |
| Ile84Ser | Yes | Yes |
| His88Arg | Yes | Yes |
| Glu89Gln (2 patients) | Yes | Yes |
| Ala97Ser | Yes | Yes |
| Tyr114Cys | Yes | Yes |
DMSO dimethylsulfoxide, FOI fraction of initial
aPercent stabilization ≥ 32%
bDMSO vehicle FOI was zero, therefore percent stabilization could not be determined
cFOI in tafamidis-spiked sample (but not DMSO-spiked sample) was quantifiable, suggesting the sample was indeed stabilized by tafamidis
dDeemed stabilized but upon retesting, did not cross 32% stabilization threshold (26% stabilization)