Drug Studies on Rett Syndrome: From Bench to Bedside.

Gomathi et al. (2020) looked at every drug trial ever run for Rett syndrome. They counted 70 different compounds. None had reached the final, large-scale test phase.

The team pulled papers from 1966 to 2019. They wanted to see which medicines moved from lab to clinic.

Zero drugs made it to phase III. Most stayed in Petri dishes or early safety tests.

The review shows a clear gap: lots of chemicals, no proven pill.

Bauman (1991) first mapped Rett features 29 years earlier. That paper described the disorder; Mohan shows the drug pipeline is still empty.

Matson et al. (2004) tried behavioral fixes for hand stereotypy and saw little change. Their null result supports Mohan’s point: if drugs and ABA both struggle, we need new science.

Galuska et al. (2006) used eye-tracking to reveal hidden cognitive skills. Mohan includes such studies to warn drug makers: pick outcome tools that can detect small gains.

Ward et al. (2021) found kids can express 1–3 words with informal eye-gaze tasks. Mohan cites this as proof that adapted measures, not standard IQ tests, should end drug trials.

For BCBAs, the message is simple: stay in your lane. No pill is coming soon. Keep teaching communication with eye-gaze devices, sensory breaks, and functional routines. Track tiny gains—the same micro-skills drug trials will need. Your data may even help future studies pick better endpoints.