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Target Validation of Splicing Repression, a Major Function of TDP-43 in the Motor Neuron

Unmet NeedAmyotrophic lateral sclerosis (ALS) is a rare but debilitating disease characterized by the death of motor neurons that eventually leads to difficulty moving, speaking, swallowing, and breathing. The fundamental cause of ALS is unknown and as a result treatment options are primarily restricted to physical therapy to help prolong muscle strength....

Published: 3/14/2025 | Inventor(s): Philip Wong, Aneesh Donde, Jonathan Ling, Liam Chen, Mingkuan Sun

Keywords(s): Amyotrophic Lateral Sclerosis, CNS and Neurological Disorders, Disease Indication, Target, Therapeutic Matter, Therapeutics

Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Therapeutic Modalities > Targets