Changes in Cerebrospinal Fluid Concentrations of Selenium Species Induced by Tofersen Administration in Subjects with Amyotrophic Lateral Sclerosis Carrying SOD1 Gene Mutations Changes in Cerebrospinal Fluid Concentrations of Selenium Species Induced by Tofersen Administration in Subjects with Amyotrophic Lateral Sclerosis Carrying SOD1 Gene Mutations

• Published in: Biological trace element research Vol. 203; no. 4; pp. 2355 - 2364
• Main Authors: Vinceti, Marco, Urbano, Teresa, Filippini, Tommaso, Bedin, Roberta, Simonini, Cecilia, Sorarù, Gianni, Trojsi, Francesca, Michalke, Bernhard, Mandrioli, Jessica
• Format: Journal Article
• Language: English
• Published: New York Springer US 01.04.2025; Springer Nature B.V
• Subjects: Adult; Aetiology; Aged; Amyotrophic lateral sclerosis; Amyotrophic Lateral Sclerosis - cerebrospinal fluid; Amyotrophic Lateral Sclerosis - drug therapy; Amyotrophic Lateral Sclerosis - genetics; Anion exchange; anion exchange chromatography; Anion exchanging; Anions; Antisense oligonucleotides; Antisense therapy; Biochemistry; Biomedical and Life Sciences; Biotechnology; Blood-brain barrier; Brain; Central nervous system; Cerebrospinal fluid; Chromatography; disease progression; Drugs; etiology; Female; Food and Drug Administration; genes; Health services; Humans; Inductively coupled plasma; Life Sciences; Male; Mass spectrometry; Mass spectroscopy; Middle Aged; Motor neurons; Mutation; Neurodegenerative diseases; Neurons; Nutrition; oligonucleotides; Oligonucleotides - administration & dosage; Oncology; Point mutation; Scientific imaging; Selenium; Selenium - cerebrospinal fluid; Selenomethionine; species; Spinal cord; Superoxide dismutase; Superoxide Dismutase-1 - genetics; toxicology; Selenium compounds; Amyotrophic lateral sclerosis; Selenium; Tofersen; SOD1

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease affecting the brain and spinal cord motor neurons. On 25 April 2023, the drug tofersen, an antisense oligonucleotide, received the US Food and Drug Administration approval for treating ALS in adults carrying mutations of the SO...