Whatever the actual reasons, the practical result is that the compound has not entered the clinical evidence pipeline
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
Sensory Symptoms Numbness, tingling, or pins and needles (paresthesia) typically distal/stocking-glove in polyneuropathy Burning pain, allodynia, hyperalgesia common in small-fiber predominant neuropathy and CIDP Loss of proprioception and vibration sense sensory ataxia, positive Romberg sign Loss of pain/temperature sensation (small fiber) risk of unnoticed wounds Motor Symptoms Distal muscle weakness (foot drop in peroneal neuropathy, wrist drop in radial neuropathy) Atrophy of intrinsic hand muscles (ulnar/median neuropathy) Hyporeflexia or areflexia (ankle jerks lost early in length-dependent polyneuropathy) Fasciculations in severe axonal loss Autonomic Symptoms Orthostatic hypotension, dizziness on standing Gastroparesis, constipation or diarrhea, nausea Neurogenic bladder (hesitancy, retention, incontinence) Anhidrosis or gustatory sweating Sexual dysfunction
Cells were trypsinised and transferred onto Matrigel on day 6 in serum-free media containing the compounds
Am J Respir Cell Mol Biol 41:50915 Cooper AJL, Hanigan MH (2010) 4.17Enzymes involved in processing glutathione conjugates