Congress: Movement Disorder Society (MDS) International Congress 2026
Dates: October 4-8, 2026
Location: Seoul, Korea — COEX Convention and Exhibition Center
The MDS 2026 congress will feature dedicated sessions on ataxias and cerebellar disorders, covering both inherited genetic ataxias and acquired cerebellar conditions. These sessions will address the latest advances in diagnosis, genotype-phenotype correlations, disease-modifying therapies, and symptomatic management approaches.
The spinocerebellar ataxias are a heterogeneous group of autosomal dominant neurodegenerative disorders characterized by progressive cerebellar ataxia, often accompanied by other neurological features including oculomotor abnormalities, peripheral neuropathy, and cognitive impairment.
- Gene: ATXN1 (CAG repeat expansion)
- Features: Ataxia, spasticity, slow saccades
- Therapeutic status: ASO clinical trials ongoing
- Gene: ATXN2 (CAG repeat expansion)
- Features: Ataxia, slow saccades, myoclonus
- Gene: ATXN3 (CAG repeat expansion)
- Features: Ataxia, spasticity, ophthalmoplegia
- Most common SCA worldwide
- Gene: CACNA1A (channelopathy)
- Features: Pure cerebellar ataxia, episodic ataxia
- Therapeutic: Calcium channel modulators under investigation
- Gene: ATXN7 (CAG repeat expansion)
- Features: Ataxia with retinal degeneration
- SCA1: ASOs targeting ATXN1 — preclinical and early clinical stages
- SCA3: ASOs targeting ATXN3 — multiple preclinical studies
- SCA2: ASOs targeting ATXN2 — proof-of-concept in models
- AAV-vector delivery: Gene silencing and replacement strategies
- CRISPR-based editing: Precise genetic correction in development
- CoQ10 supplementation: For mitochondrial function support
- Physical therapy: Balance and gait training
- Speech therapy: For dysarthria management
Friedreich's ataxia (FA) is an autosomal recessive disorder caused by GAA repeat expansions in the FXN gene, leading to frataxin deficiency and mitochondrial dysfunction. It is the most common inherited ataxia with onset typically in adolescence.
- Progressive gait and limb ataxia
- Dysarthria
- Loss of deep tendon reflexes
- Cardiomyopathy (in >50% of patients)
- Diabetes mellitus (in ~30% of patients)
- Scoliosis
- Gene therapy: AAV-based FXN replacement in preclinical development
- Protein replacement: Frataxin analogs in early-stage trials
- CoQ10 + vitamin E: Combination therapy trials
- Idebenone: Antioxidant therapy (approved in some regions)
- EPI-743 (vatiquinone): Phase 2/3 trials ongoing
- Novel antioxidant compounds
- Gene therapy updates
- Cardiac outcome measures in FA trials
The episodic ataxias are channelopathies characterized by recurrent ataxic episodes with complete or partial recovery between episodes.
- Gene: KCNA1 (potassium channel)
- Features: Brief episodes (seconds to minutes), myokymia
- Treatment: Acetazolamide, carbamazepine
- Gene: CACNA1A (calcium channel)
- Features: Longer episodes (hours to days), ataxia, dysarthria
- Treatment: Acetazolamide, 4-aminopyridine
- Channel blockers: Targeted small molecules
- Gene therapy: For severe EA2 cases
- Anti-Yo, anti-Hu, anti-Tr antibodies
- Underlying oncology identification
- Immunotherapy approaches
- Chronic alcohol consumption as risk factor
- Thiamine supplementation strategies
- Gluten ataxia
- Opsoclonus-myoclonus syndrome
- Tandem walking evaluation
- Dynamic gait index
- Computerized gait analysis
- Intensive balance training
- Gait-specific physical therapy
- Assistive devices (canes, walkers)
- Amantadine (controversial benefit)
- 4-Aminopyridine for cerebellar ataxia
- Buspirone (serotonergic modulation)
- Deep brain stimulation for select cases
- Cerebellar stimulation trials ongoing
- ATM gene mutations
- Immunodeficiency and cancer risk
- Novel therapeutic targets
- AOA1 (APT1 gene)
- AOA2 (SETX gene)
- Typical onset in childhood
- Serum neurofilament light chain (NfL) for disease progression
- Imaging biomarkers (MRI volumetric measures)
- Genetic testing standardization
- Natural history study harmonization
- Endpoint standardization (SARA, ICARS scales)
- Biomarker-guided patient selection
- RNA therapeutics: ASOs and siRNA for genetic ataxias
- Cell-based therapies: Neural stem cell transplantation
- Neuroprotective compounds: Mitochondrial stabilizers