Friedreich ataxia: clinical features and new developments

The multifaceted approach for clinical care in FRDA has necessitated the development of disease-specific clinical care guidelines. New developments in FRDA include the advancement of clinical drug trials targeting the NRF2 pathway and frataxin restoration. Additionally, a novel understanding of gene silencing in FRDA, reflecting a variegated silencing pattern, will have applications to current and future therapeutic interventions. Finally, new perspectives on the neuroanatomy of FRDA and its developmental features will refine the time course and anatomical targeting of novel approaches.

Read the Full article here
 


SHARE
 
kyle-friend-eb.jpg
Privacy Policy      Service Terms      Contact      Charity Navigator