Rett syndrome
- Trofinetide is approved for core Rett symptoms
- Multidisciplinary therapy remains essential
- Treat seizures, scoliosis, feeding + breathing problems
- PT/OT/speech/communication support function
- GI adverse effects can limit trofinetide
- Therapy improves symptoms—not the underlying mutation
Use at a glance
- Trofinetide is approved for core Rett symptoms
Treatment options
- Multidisciplinary therapy remains essential
- Treat seizures, scoliosis, feeding + breathing problems
Other treatment options
- GI adverse effects can limit trofinetide
- Therapy improves symptoms—not the underlying mutation
Practical pearls
- PT/OT/speech/communication support function
Self-check
- Does Rett treatment change the underlying mutation?
No—it improves symptoms. - What can limit trofinetide in Rett syndrome?
GI adverse effects.
Drugs used for this (1)
All clinical uses · Open Rett syndrome in the interactive console ↗