Advances in Neurological Rare Disease Therapeutic Research


These are exciting times for neurological disease research and the discovery of new therapeutic strategies for better treatment. In this webinar, Dr. Muñoz-Sanjuan discussed the current state of therapeutic programs and efforts of the CHDI Foundation aiming to silence the mutant Huntingtin gene. Dr. Ozdinler closed the session by presenting the exciting identification of NU-9, the first compound shown to improve the health of upper motor neurons, whose degeneration leads to many neurological disorders including ALS, HSP, and PLS.