Trials & Treatments Reporting
ALS Nexus 2026: Experimental therapy shows early promise in SOD1-ALS
Relevant to people following emerging treatment options for SOD1-ALS
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ResearchHuman
Butyrate reduced ALS-linked protein clumps and improved movement in worms and cells
Directly informs HDAC inhibition, SOD1 aggregation and ALS model research
Trials & TreatmentsHumanMouse
JP1 improved movement and survival in ALS model mice by targeting oxidative stress and autophagy
Provides mechanistic and efficacy data on JP1, Nrf2 signaling, autophagy and ALS mouse models.
Trials & Treatments
A severe hypersensitivity reaction was reported during tofersen therapy in ALS
Important for clinicians assessing tofersen hypersensitivity and treatment safety
ResearchHuman
Researchers identify a signaling pathway that controls ADH5/GSNOR in SOD1 ALS cell models
Defines a redox-dependent ATM-CHK2-SOD1 pathway and identifies ADH5/GSNOR as a potential target.
ResearchAnimal
A guinea pig pregnancy study found no measurable benefit from dimethyl fumarate
Provides a guinea-pig pregnancy model and maternal-fetal transcriptomic data for Nrf2 and DMF research.
ResearchHuman
Tofersen treatment did not lower blood SOD1 activity in a study of people with SOD1-related ALS
Mainly of interest to researchers studying SOD1 activity, mutation effects and tofersen mechanisms.
ResearchHuman
A Chinese study describes clinical and genetic differences in young-onset ALS
Mainly of interest to researchers studying age-dependent ALS genetics and trial design.
ResearchMouse
Researchers generated mouse models carrying single-copy mutant human SOD1 genes
Directly relevant to researchers studying SOD1 models and genome-editing therapy evaluation.
Trials & TreatmentsHumanMouseUpdated
ALS Untangled does not recommend ivermectin for ALS
Relevant to clinicians advising on ivermectin evidence, safety and lack of ALS trials
ResearchHumanMouse
Neural stem cell vesicles slowed disease progression in an ALS mouse model
Directly informs extracellular-vesicle therapy, motor-neuron protection and p53-pathway research.
ResearchHuman
A Brazilian study found pathogenic gene variants in 30 people with motor neuron disease
Mainly of interest to researchers studying MND genetics, testing patterns, and population variation.
ResearchCase reportHuman
Four people with SOD1 D91A ALS first developed progressive ataxia
Mainly of interest to researchers studying SOD1 genotype–phenotype relationships and diagnostic latency.
ResearchAnimal
Paternal lead exposure caused multigenerational oxidative changes in fruit flies
Provides transgenerational oxidative-stress and antioxidant-gene findings for researchers studying toxicology and disease mechanisms.
Trials & TreatmentsHuman
A study mapped SOD1 variants and tofersen access in six Central Eastern European countries
Relevant to clinicians assessing SOD1 variants, genetic testing, counseling, and tofersen access.
ResearchReviewHuman
Brazilian experts recommend early genetic testing for people with ALS
Relevant to clinicians updating ALS genetic-testing practice and counselling
ResearchHuman
A Lithuanian study found pathogenic genetic variants in 15.7% of patients with motor neuron disease
Mainly of interest to researchers studying MND genetics and regional variation
Trials & TreatmentsHuman
Tofersen was linked to slower decline in one person with ALS and an uncertain SOD1 variant
Relevant to clinicians assessing tofersen for SOD1 variants of uncertain significance and monitoring biomarkers.
Trials & TreatmentsHuman
A French real-world study links tofersen with slower decline in SOD1-ALS
Relevant to clinicians evaluating real-world tofersen effectiveness, biomarkers, survival and study limitations.
Trials & TreatmentsMouse
Borsantrazole extended survival in a mouse model of ALS
Mainly of interest to researchers studying oxidative-stress therapeutics, proteomics and ALS models.
ResearchAnimal
A canine ALS-like disease study links sensory pathway damage with walking problems
Important for researchers studying SOD1 pathology, sensory pathways and disease-modifying factors in a canine model.
Trials & TreatmentsHumanMouse
Platelet factor 4 improved disease measures in SOD1 ALS mice
Mainly of interest to researchers studying PF4, OPTN-dependent autophagy and SOD1 pathology
ResearchHumanMouse
A perspective calls for broader ALS models beyond protein-focused mouse studies
Relevant to researchers studying protein pathology, transgenic models, and translational ALS methods.
ResearchCommentary
A case report describes early severe chewing-muscle involvement in SOD1-associated ALS
Mainly of interest to researchers studying SOD1-ALS phenotypes and biomarker-clinical dissociation.
Trials & TreatmentsHuman
Macrophage inclusions were more common in the cerebrospinal fluid of patients receiving tofersen than nusinersen
Relevant to clinicians interpreting macrophage inclusions, inflammation and treatment response in CSF.
ResearchHuman
Sponsored genetic testing found disease-causing variants in 12.9% of 170 people with ALS
Mainly of interest to researchers studying ALS genetics and testing strategies
Trials & TreatmentsHuman
A U.S. center reports delivering tofersen to 11 people with SOD1 ALS before approval
Relevant to clinicians implementing tofersen expanded access and assessing operational requirements
ResearchCase reportHuman
A SOD1 mutation was linked to protein instability in a rapidly progressing ALS case
Offers molecular dynamics evidence about N87D-driven SOD1 instability and aggregation
ResearchHumanComputational
The supplied study concerns Alzheimer’s disease rather than ALS or MND
Relevant to researchers comparing SOD1 and mitochondrial mechanisms across neurodegenerative diseases
ResearchHuman
UK Biobank analysis finds more pathogenic SOD1 carriers than expected from clinical reports
Mainly of interest to researchers studying SOD1 penetrance, prevalence and biomarkers.
ResearchObservational studyHuman
A CSF protein showed an early response to tofersen in people with SOD1-ALS
Mainly of interest to researchers studying ALS biomarkers, proteomics and targeted therapy response
ResearchHumanMouse
Different SOD1 mutations produced faster or slower disease patterns in mice
Directly informs research on misfolded SOD1 strains, seeding and disease progression.
Research Compass
A phase 2 trial of RAG-17 has begun for SOD1-related ALS
Mainly of interest to researchers tracking a broad set of ALS mechanisms, biomarkers and interventions
ResearchReviewHumanAnimal
A review examines how ubiquitin markings influence protein buildup in ALS
Summarises ubiquitin regulation of ALS-associated protein aggregation and degradation pathways
Research CompassMainly relevant to Canada.
Qalsody moves closer to public funding in Canada
Mainly of interest to researchers surveying current ALS biomarkers, mechanisms, trials, and policy signals.
ResearchCase reportHuman
Researchers identified a new SOD1 variant in a Japanese family with familial ALS
Mainly of interest to researchers studying SOD1 structure, pathology, and genotype-phenotype relationships.
ResearchHuman
N-terminal acetylation speeds amyloid formation by SOD1 in a laboratory study
Directly informs research on SOD1 post-translational modification, amyloid nucleation and fibril growth.
ResearchHumanMouse
Researchers identified distinct fibril structures formed by two ALS-linked SOD1 variants
Directly informs SOD1 aggregation structures, mutant-specific toxicity and ALS disease modelling.
Research
A mouse study identifies a disease-associated motor neuron state before cell death
Provides longitudinal single-cell and spatial molecular data on motor-neuron vulnerability and glial interactions.
ResearchHumanMouse
An experimental siRNA treatment extended survival in SOD1 ALS mice
Mainly of interest to researchers studying siRNA delivery and SOD1 disease models
Trials & Treatments
International clinical trial updates to look out for in 2025
Relevant to clinicians monitoring upcoming efficacy and safety evidence
Research
Pre-symptomatic detection and early diagnosis in MND
Mainly of interest to researchers studying presymptomatic disease and prevention trial design
ResearchHuman
CAPRIN1 condensates shift immature SOD1 toward unfolding and aggregation
Directly informs condensate biology, SOD1 folding, aggregation and ALS mutation mechanisms
Research
How are proteins made and what happens when they go wrong in MND?
Summarises gene-to-protein processing and disease-linked SOD1 and TDP-43 mechanisms
ResearchHuman
Phialomustin-B reduced SOD1 aggregation in laboratory studies
Provides structural and aggregation data on a potential SOD1-modulating lead compound.
ResearchReviewHumanAnimal
A review links high MSG exposure to proposed mechanisms of neurodegeneration
Relevant to researchers examining excitotoxicity, disease mechanisms and blood-brain-barrier-penetrant phytochemicals.
Advocacy
Reuniting the MND community at the 34th International Symposium on ALS/MND
Mainly of interest to researchers following new mechanisms, biomarkers, trials and collaborations.
ResearchAnimal
The cyanotoxin 2,4-DAB caused toxic effects in developing zebrafish
Provides zebrafish toxicity, behavioral, proteomic and ALS-associated gene findings for neurodegeneration research.
ResearchHumanMouse
SRCP1 reduced some aggregated SOD1 protein in ALS models but did not extend mouse survival
Provides mechanistic and model-comparison data on SRCP1, SOD1 aggregation and therapeutic context dependence.
ResearchHuman
A cell study linked toxic SOD1 forms to motor neuron-like cell death
Provides mechanistic evidence distinguishing toxic SOD1 trimers from protective large aggregates.
Trials & Treatments
ALS Untangled does not recommend CuATSM for ALS outside clinical trials
Relevant to clinicians advising on CuATSM evidence, dosing uncertainty and safety.
ResearchHumanMouse
Improved mitochondrial calcium buffering did not extend survival in ALS mice
Provides mechanistic evidence that mitochondrial calcium buffering affects pathology but not paralysis or survival in SOD1 mouse models.