Table 3. Positive (+) or no response (−) of FL-SMN mRNA and SMN protein in lymphoblasts after treatment with HU, VPA and PBA.
HU | VPA | PBA | ||||
---|---|---|---|---|---|---|
Samples | FL-SMN | Prot | FL-SMN | Prot | FL-SMN | Prot |
1 | − | − | + | − | − | − |
2 | − | − | − | + | − | − |
3 | + | − | + | − | + | − |
4 | + | − | + | − | − | − |
5 | − | − | − | − | − | − |
6 | − | − | − | − | − | − |
7 | + | − | − | − | − | − |
No categorical responders (positive in mRNA and protein) were observed, only non-responders (negative in mRNA and protein) or partial responders (discordance between mRNA and protein).