Management

Treatment of the manifestations and supportive care are recommended and can be managed by the appropriate specialty.

Fingolimod (FTY720)
In zebrafish model, the enzymatic inhibition of Cer synthase by fingolimod, 1 step prior to DEGS1 in the pathway, reduced the critical DhCer/ Cer imbalance and the severe locomotor disability, increasing the number of myelinating oligodendrocytes. Therefore, Fingolimod (FTY720) (or novel, more specific next-generation sphingosine analogs interfering with CerS or other enzymatic steps along the pathway) may provide a promising therapeutic option for white-matter disorders linked to DEGS1 deficiency.