Clinical Study

IGF1 as a Potential Treatment for Rett Syndrome: Safety Assessment in Six Rett Patients

Table 1

Selected patients for the study. This table reports the patients in the study, with their specific mutation, D0 and age at the beginning of the treatment. In addition, it reports the gravity of the disease (7 heavily impaired, 1 mildly impaired).

IDD0 (Age)MutationSeverity (1–7)

S14.8Del exons 3 and 44
S28.1T158M (missense)6
S35.11R133C (missense)3
S45.9C13ins in GCCGC in exon 1 (frameshift)3
S54.7R270X (nonsense)5
S610.81055 del 12 + 1157 del 44 (frameshift)3