Experts discuss hurdles and prospects for the first approved Fragile X therapy

In a recent episode of BioSpace's "Denatured" podcast, Hilary Rosselot of the National Fragile X Foundation and Jordi Fàbrega, co‑founder and CEO of CONNECTA Therapeutics, talked about the current state of Fragile X research. They highlighted the difficulty of defining credible clinical trial endpoints and the persistent challenge of placebo effects in studies.

The conversation also explored emerging scientific approaches, notably neuroplasticity modulation, as a potential way to overcome these obstacles. Both guests emphasized the need for robust trial designs and patient‑focused strategies to build a sustainable path toward an approved treatment.

While no new drug data were presented, the discussion underscored the broader industry effort to move Fragile X from a research bottleneck to a therapeutic reality, signaling optimism tempered by the practical challenges that remain.