Proteostasis Therapeutics, Inc. is using a novel approach to treat orphan and neurodegenerative diseases, including ALS, by regulating protein homeostasis. The company recently presented their latest findings at multiple scientific conferences, including the Alzheimer’s Association International Conference (AAIC). At AAIC, the company showed that their promising lead compound could decrease toxic protein aggregations in neurons, and rescue the neurons from cell death. In certain cases of ALS, the ALS-associated protein, TDP-43, accumulates inside of neurons in protein aggregates. Proteostasis’ approach to target clearance of such aggregates might rescue these neurons from death and may be a viable therapy for ALS patients.
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