INTERMEDIATE-SIZED EXPANDED ACCESS PROTOCOL FOR CNM-AU8 IN AMYOTROPHIC LATERAL SCLEROSIS (ALS). - ABSTRACT COLUMBIA UNIVERSITY, CLENE, AND SYNAPTICURE ARE PARTNERING AND TAKING AN INNOVATIVE APPROACH TO PROVIDE PERSONS LIVING WITH AMYOTROPHIC LATERAL SCLEROSIS ALS (PALS) ACROSS ALL 50 STATES— INCLUDING THOSE IN REMOTE/RURAL AREAS—ACCESS TO CNM-AU8®, A WELL-TOLERATED NEUROPROTECTIVE DRUG BEING INVESTIGATED FOR TREATMENT OF ALS. ALS IS A PROGRESSIVE NEURODEGENERATIVE DISEASE AFFECTING MOTOR NEURONS OF THE BRAIN AND SPINAL CORD. INVESTIGATIONS OF THE MECHANISM OF THIS DISEASE HAVE REVEALED THAT MOTOR NEURONS ARE ENERGETICALLY IMPAIRED IN ALS. SIGNS OF ENERGETIC IMPAIRMENT IN MOTOR NEURONS PRECEDE CLINICAL MANIFESTATIONS, AND ENERGETIC IMPAIRMENT IS KEY TO THE EVENTS AFFECTING MITOCHONDRIAL FUNCTION, GLUTAMATERGIC SIGNALING, CALCIUM HOMEOSTASIS, RNA PROCESSING/FUNCTION, AND PROTEOSTASIS, LEADING TO NEURONAL DEATH. CNM-AU8 IS AN ORALLY ADMINISTERED SUSPENSION OF BLOOD-BRAIN BARRIER PENETRAN

Summary

INTERMEDIATE-SIZED EXPANDED ACCESS PROTOCOL FOR CNM-AU8 IN AMYOTROPHIC LATERAL SCLEROSIS (ALS). - ABSTRACT COLUMBIA UNIVERSITY, CLENE, AND SYNAPTICURE ARE PARTNERING AND TAKING AN INNOVATIVE APPROACH TO PROVIDE PERSONS LIVING WITH AMYOTROPHIC LATERAL SCLEROSIS ALS (PALS) ACROSS ALL 50 STATES— INCLUDING THOSE IN REMOTE/RURAL AREAS—ACCESS TO CNM-AU8®, A WELL-TOLERATED NEUROPROTECTIVE DRUG BEING INVESTIGATED FOR TREATMENT OF ALS. ALS IS A PROGRESSIVE NEURODEGENERATIVE DISEASE AFFECTING MOTOR NEURONS OF THE BRAIN AND SPINAL CORD. INVESTIGATIONS OF THE MECHANISM OF THIS DISEASE HAVE REVEALED THAT MOTOR NEURONS ARE ENERGETICALLY IMPAIRED IN ALS. SIGNS OF ENERGETIC IMPAIRMENT IN MOTOR NEURONS PRECEDE CLINICAL MANIFESTATIONS, AND ENERGETIC IMPAIRMENT IS KEY TO THE EVENTS AFFECTING MITOCHONDRIAL FUNCTION, GLUTAMATERGIC SIGNALING, CALCIUM HOMEOSTASIS, RNA PROCESSING/FUNCTION, AND PROTEOSTASIS, LEADING TO NEURONAL DEATH. CNM-AU8 IS AN ORALLY ADMINISTERED SUSPENSION OF BLOOD-BRAIN BARRIER PENETRAN