MDS 2026 — Ataxia and Cerebellar Disorders

Congress: Movement Disorder Society (MDS) International Congress 2026 Dates: October 4-8, 2026 Location: Seoul, Korea — COEX Convention and Exhibition Center

Overview

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["@mds"].

Spinocerebellar Ataxias (SCAs)

Overview

Congress: Movement Disorder Society (MDS) International Congress 2026 Dates: October 4-8, 2026 Location: Seoul, Korea — COEX Convention and Exhibition Center

Overview

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["@mds"].

Spinocerebellar Ataxias (SCAs)

Overview

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[@klockgether2018].

Major SCA Subtypes

SCA1 (ATXN1)

• Gene: ATXN1 (CAG repeat expansion)
• Features: Ataxia, spasticity, slow saccades
• Therapeutic status: ASO clinical trials ongoing

SCA2 (ATXN2)

• Gene: ATXN2 (CAG repeat expansion)
• Features: Ataxia, slow saccades, myoclonus

SCA3 (ATXN3) — Machado-Joseph Disease

• Gene: ATXN3 (CAG repeat expansion)
• Features: Ataxia, spasticity, ophthalmoplegia
• Most common SCA worldwide

SCA6 (CACNA1A)

• Gene: CACNA1A (channelopathy)
• Features: Pure cerebellar ataxia, episodic ataxia
• Therapeutic: Calcium channel modulators under investigation

SCA7

• Gene: ATXN7 (CAG repeat expansion)
• Features: Ataxia with retinal degeneration

Emerging Therapies

Antisense Oligonucleotides (ASOs)

• 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

Gene Therapy Approaches

• AAV-vector delivery: Gene silencing and replacement strategies
• CRISPR-based editing: Precise genetic correction in development

Symptomatic Management

• CoQ10 supplementation: For mitochondrial function support
• Physical therapy: Balance and gait training
• Speech therapy: For dysarthria management[@ashida2021]

Friedreich's Ataxia

Overview

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[@martindale2022].

Clinical Features

• Progressive gait and limb ataxia
• Dysarthria
• Loss of deep tendon reflexes
• Cardiomyopathy (in >50% of patients)
• Diabetes mellitus (in ~30% of patients)
• Scoliosis

Disease-Modifying Therapies

Frataxin Restoration

• Gene therapy: AAV-based FXN replacement in preclinical development
• Protein replacement: Frataxin analogs in early-stage trials

Mitochondrial Function

• CoQ10 + vitamin E: Combination therapy trials
• Idebenone: Antioxidant therapy (approved in some regions)
• EPI-743 (vatiquinone): Phase 2/3 trials ongoing

Clinical Trials at MDS 2026

• Novel antioxidant compounds
• Gene therapy updates
• Cardiac outcome measures in FA trials

Episodic Ataxias

Overview

The episodic ataxias are channelopathies characterized by recurrent ataxic episodes with complete or partial recovery between episodes.

Major Types

EA1 (KCNA1)

• Gene: KCNA1 (potassium channel)
• Features: Brief episodes (seconds to minutes), myokymia
• Treatment: Acetazolamide, carbamazepine

EA2 (CACNA1A)

• Gene: CACNA1A (calcium channel)
• Features: Longer episodes (hours to days), ataxia, dysarthria
• Treatment: Acetazolamide, 4-aminopyridine

Novel Therapies

• Channel blockers: Targeted small molecules
• Gene therapy: For severe EA2 cases

Cerebellar Degeneration: Non-Genetic Causes

Paraneoplastic Cerebellar Degeneration

• Anti-Yo, anti-Hu, anti-Tr antibodies
• Underlying oncology identification
• Immunotherapy approaches

Alcohol-Related Cerebellar Degeneration

• Chronic alcohol consumption as risk factor
• Thiamine supplementation strategies

Autoimmune Ataxias

• Gluten ataxia
• Opsoclonus-myoclonus syndrome

Gait Ataxia

Clinical Assessment

• Tandem walking evaluation
• Dynamic gait index
• Computerized gait analysis

Therapeutic Approaches

Rehabilitation

• Intensive balance training
• Gait-specific physical therapy
• Assistive devices (canes, walkers)

Pharmacologic

• Amantadine (controversial benefit)
• 4-Aminopyridine for cerebellar ataxia
• Buspirone (serotonergic modulation)

Surgical

• Deep brain stimulation for select cases
• Cerebellar stimulation trials ongoing

Pediatric Ataxias

Ataxia-Telangiectasia

• ATM gene mutations
• Immunodeficiency and cancer risk
• Novel therapeutic targets

Ataxia with Oculomotor Apraxia (AOA)

• AOA1 (APT1 gene)
• AOA2 (SETX gene)
• Typical onset in childhood

Research Priorities at MDS 2026

Biomarkers

• Serum neurofilament light chain (NfL) for disease progression
• Imaging biomarkers (MRI volumetric measures)
• Genetic testing standardization

Clinical Trial Design

• Natural history study harmonization
• Endpoint standardization (SARA, ICARS scales)
• Biomarker-guided patient selection

Emerging Modalities

• RNA therapeutics: ASOs and siRNA for genetic ataxias
• Cell-based therapies: Neural stem cell transplantation
• Neuroprotective compounds: Mitochondrial stabilizers

Related Pages

• [MDS 2026 — Rare Movement Disorders](/events/mds-2026-rare-movement-disorders)](/events)
• [MDS 2026 — Main Congress Page](/events/mds-2026)](/events)
• [Spinocerebellar Ataxia Type 1](/diseases/spinocerebellar-ataxia-type-1)](/diseases/cerebellar-ataxia)
• [Spinocerebellar Ataxia Type 2](/diseases/spinocerebellar-ataxia-type-2)](/diseases/cerebellar-ataxia)
• [Friedreich's Ataxia](/diseases/friedreich-ataxia)
• [Ataxia-Telangiectasia](/diseases/ataxia-telangiectasia)
• [Cerebellar Ataxia](/diseases/cerebellar-ataxia)

References