Session 16 - The changing panorama of treatment

Chairs:
P. Huppke, NN

Themen:

• RNA Editing
• Update MeCP2 reactivation
• Trofinetide – first approved medication to treat Rett syndrome (J. Neul)
• Update Gene Therapy and preliminary results (Taysha)
• Update Gene Therapy and preliminary results (Neurogene)