NGN‑401 · Embolden

Another gene approach, designed to control MECP2 expression.

Study level

The aim, simply

The pivotal Embolden study evaluates the effects, safety and tolerability of NGN‑401, building on the initial phase 1/2 programme.

Criteria: guidance, not eligibility

The pivotal cohort lists age 3 or older, typical Rett with a MECP2 mutation and a post-regression stage. This is not the full set of criteria.

What this cannot tell us

A study record describes research, not a guaranteed benefit or treatment access in your country.

Only the trial team can confirm eligibility. Age and country are not enough: the protocol includes other criteria. Status may also vary by study site.

Centre level

Partial list. A registry listing does not confirm a place. Check directly with the site.