Different genetic mutations can converge on the same downstream pathology. The C9ORF72 repeat expansion and progranulin haploinsufficiency both trigger the RNA-binding protein TDP-43 to abandon the ...
The TDP-43 RNA-binding protein is found in abnormal cytoplasmic inclusions in affected motor neurons in almost all ALS cases, and mutations in the TDP-43 gene (TARDBP) cause a rare form of familial ...
VectorY Therapeutics Receives FDA Clearance of IND to Proceed with the PIONEER-ALS Phase 1/2 Trial of VTx-002, a First-in-Class Vectorized Antibody Targeting TDP-43 Pathology in ALS VectorY ...
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