第61回日本神経学会学術大会

講演情報

ホットトピックス

[HT-17] ホットトピックス17
次々に実現する神経筋疾患の核酸医薬の今後の展望

2020年9月2日(水) 10:45 〜 12:15 第01会場 (岡山コンベンションセンター 4F 405会議室)

座長:横田 隆徳(東京医科歯科大学大学院医歯学総合研究科脳神経病態学分野),戸田 達史(東京大学大学院医学系研究科神経内科学)

[HT-17-1] Clinical application siRNA for neurological diseases

Muthiah Manoharan (Alnylam Pharmaceuticals, Inc.)

Following antisense oligonucleotide, nusinersen/Spinraza for SMA, first siRNA drug, givosiran/Fitusiran for TTR amyloidosis (familial amyloid neuropathy) has been approved in Japan, and SOD1 and C9ORF72 for ALS, tau for Alzheimer disease, PSP/CBD, synuclein for Parkinson disease, DLB and MSA, and Huntinting for Huntington disease are also started for clinical stages. This rapid and splendid progress of oligonucleotide therapy are now a main stream for development of DMT to neurological diseases. In this symposium, basic chemistry, delivery and biology of oligonucleotide drug are reviewed, and current clinical status for approved oligonucleotide drugs for neurological diseases are to be updated.