Division of Rare / Intractable Disease Research

• 04/06/20
  Safe thrombolytic therapy with alteplase for cerebral infarction of unknown onset time without the FLAIR imaging findings of cerebral infarction
• 03/27/20
  Marketing approval of NS-065/NCNP-01 (“viltolarsen”) for the treatment of Duchenne muscular dystrophy
• 03/19/20
  Identification of an intracellular signal critical for the development of head and neck cancer - Creation of a mouse model for the fastest cancer development -
• 03/13/20
  Identification of the gene responsible for progressive myoclonic epilepsy
• 03/06/20
  Elucidation of the genetic mechanism of efficient DNA damage repair - Revelation of the pathogenesis of Cockayne syndrome (progeria), a rare hereditary intractable disease/Establishment of a disease mouse model useful for elucidating the pathological conditions of various aging-r...
• 02/19/20
  Important roles of the synapses in spinal and bulbar muscular atrophy - Elucidation of pathology and development of therapies by targeting neuromuscular junctions -
• 02/06/20
  Results from a research on the prognosis of Leigh syndrome in Japan
• 02/06/20
  Semaphorin is a key molecule that induces the formation of nasal polyps: A novel diagnostic and treatment target for refractory empyema
• 01/27/20
  Visual restoration by innovative gene therapy using genome editing technologies – toward realization of Gene therapy to normalize gene mutations -
• 01/17/20
  Elucidation of intestinal environment changes due to gastrectomy - Overcoming complications after gastrectomy