Background：Amyotrophic lateral sclerosis, or ALS, is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. Some cases are inherited.

ALS often begins with muscle twitching and weakness in a limb, or slurred speech. Eventually, ALS affects control of the muscles needed to move, speak, eat and breathe. There is no cure for this fatal disease.

Model: TDP-43 induced ALS

Standard study duration: 12-16 weeks

Clinical endpoint:

•     Clinical scoring of neuro-deficit

•     Nerve conduction and needle EMG assay

•     Object retrieval test

•     Behavioral analysis

•     Muscle atrophy quantification by MRI

•     Pathological examinations

Features: overexpression of wild-type human transactive response deoxyribonucleic acid-binding protein 43 in spinal cords, human-similar manifestations such as motor degeneration and muscle atrophy.

Status: Open for Co-development