PMID-sentid Pub_year Sent_text compound_name comp_offset prot_official_name organism prot_offset 34704267-3 2022 Recently, the results of a phase I/II study using the antisense oligonucleotides Tofersen to treat familial amyotrophic lateral sclerosis with superoxide dismutase 1 mutation have been reported. tofersen 81-89 superoxide dismutase 1 Homo sapiens 143-165 32640130-2 2020 Intrathecal administration of tofersen is being studied for the treatment of amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. tofersen 30-38 superoxide dismutase 1 Homo sapiens 120-124 32640130-13 2020 CONCLUSIONS: In adults with ALS due to SOD1 mutations, CSF SOD1 concentrations decreased at the highest concentration of tofersen administered intrathecally over a period of 12 weeks. tofersen 121-129 superoxide dismutase 1 Homo sapiens 39-43 32640130-13 2020 CONCLUSIONS: In adults with ALS due to SOD1 mutations, CSF SOD1 concentrations decreased at the highest concentration of tofersen administered intrathecally over a period of 12 weeks. tofersen 121-129 superoxide dismutase 1 Homo sapiens 59-63 34704267-4 2022 Intrathecal Tofersen administration resulted in a 36% reduction in superoxide dismutase 1 level in the cerebrospinal fluid. tofersen 12-20 superoxide dismutase 1 Homo sapiens 67-89 35585374-0 2022 Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study. tofersen 61-69 superoxide dismutase 1 Homo sapiens 109-113 35585374-6 2022 Tofersen is an investigational antisense oligonucleotide designed to reduce synthesis of superoxide dismutase 1 (SOD1) protein through degradation of SOD1 mRNA. tofersen 0-8 superoxide dismutase 1 Homo sapiens 89-111 35585374-6 2022 Tofersen is an investigational antisense oligonucleotide designed to reduce synthesis of superoxide dismutase 1 (SOD1) protein through degradation of SOD1 mRNA. tofersen 0-8 superoxide dismutase 1 Homo sapiens 113-117 35585374-6 2022 Tofersen is an investigational antisense oligonucleotide designed to reduce synthesis of superoxide dismutase 1 (SOD1) protein through degradation of SOD1 mRNA. tofersen 0-8 superoxide dismutase 1 Homo sapiens 150-154 35585374-7 2022 Informed by Pre-fALS and the tofersen clinical development program, the ATLAS study (NCT04856982) is designed to evaluate the impact of initiating tofersen in presymptomatic carriers of SOD1 variants associated with high or complete penetrance and rapid disease progression who also have biomarker evidence of disease activity (elevated plasma NfL). tofersen 147-155 superoxide dismutase 1 Homo sapiens 186-190 35585374-7 2022 Informed by Pre-fALS and the tofersen clinical development program, the ATLAS study (NCT04856982) is designed to evaluate the impact of initiating tofersen in presymptomatic carriers of SOD1 variants associated with high or complete penetrance and rapid disease progression who also have biomarker evidence of disease activity (elevated plasma NfL). tofersen 147-155 neurofilament light chain Homo sapiens 344-347