Cerebellar & Ataxia Disorders

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Featured Session · 14 of 35

Cerebellar & Ataxia Disorders

Hereditary and acquired ataxias have a rapidly growing therapeutic pipeline with multiple disease-specific approvals. The session covers spinocerebellar ataxia (SCA) genetics, Friedreich ataxia (FA) and omaveloxolone (Skyclarys), gluten ataxia and gluten-free diet response, paraneoplastic cerebellar degeneration, multiple system atrophy cerebellar type, and emerging gene therapies for SCA. Discussion addresses cerebellar imaging including MR spectroscopy and DAT-SPECT, episodic ataxia genetics and management, cerebellar cognitive affective syndrome, ataxia-telangiectasia and DNA-repair disorders, and the international ataxia registries (CRC-SCA, GEM-NPA) driving natural-history research.

Topics covered in this session
  • SCA genetics
  • Omaveloxolone (Skyclarys) for FA
  • Gluten ataxia
  • Paraneoplastic cerebellar degeneration
  • MSA cerebellar type
  • Gene therapies for SCA
  • Cerebellar imaging
  • Ataxia-telangiectasia