Explore the molecular changes in Rett syndrome. Find out how this breakthrough could lead to more effective treatments.

Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares. Last week, the company reported that NGN-401 had shown ...

Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share price.

“We look forward to working with the EMA to address this unmet need in the hopes of potentially bringing this therapy to families who are impacted by Rett syndrome.” The application is based ...

Rett syndrome is a rare genetic disorder that affects brain development, resulting in severe mental and physical disability ... to evaluate the NGN-401 gene therapy for the treatment of female ...

If approved, trofinetide would be the first sanctioned therapy for Rett syndrome in the European Union (EU), a condition currently without specific treatment options in this region. The ...

ProQR Therapeutics has announced an $8.1 million funding boost from the Rett Syndrome Research Trust, raising the total investment to $9.1 million to advance their RNA editing collaboration.

If approved, trofinetide would be the first sanctioned therapy for Rett syndrome in the European Union (EU), a condition currently without specific treatment options in this region. The application is ...