Trofinetide

Unassigned

New Medicines

Rett syndrome in adolescent and adult females

Information

New molecular entity
Neuren Pharmaceuticals
Neuren Pharmaceuticals

Development and Regulatory status

None
None
Phase III Clinical Trials
Yes
Yes

Category

A novel synthetic analogue of the amino‐terminal tripeptide of insulin-like growth factor 1 (IGF-1)
According to European data, the prevalence of Rett syndrome is estimated to be 10 in 100,000 people with a 5 in 100,000 birth prevalence [1].
Rett syndrome in adolescent and adult females
Oral