Nusinersen treatment in adult patients with spinal muscular atrophy: a safety analysis of laboratory parameters

[1] Alves CRR(2020)Serum creatinine is a biomarker of progressive denervation in spinal muscular atrophy Neurology 94 e921-e931

[2] Zhang R(2010)RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform Annu Rev Pharmacol Toxicol 50 259-293

[3] Johnstone AJ(2018)Inotersen treatment for patients with hereditary transthyretin amyloidosis N Engl J Med 379 22-31

[4] Garner R(2019)Adult CSF total protein: higher upper reference limits should be considered worldwide. A web-based survey J Neurol Sci 396 48-51

[5] Nwe PH(2019)Adult CSF total protein upper reference limits should be age-partitioned and significantly higher than 0.45 g/L: a systematic review J Neurol 266 616-624

[6] Siranosian JJ(2006)Antisense oligonucleotides: from design to therapeutic application Clin Exp Pharmacol Physiol 33 533-540

[7] Swoboda KJ(2016)Integrated safety assessment of 2'-O-methoxyethyl chimeric antisense oligonucleotides in nonhuman primates and healthy human volunteers Mol Ther 24 1771-1782

[8] Bennett CF(2018)The effects of 2'-O-methoxyethyl oligonucleotides on renal function in humans Nucleic Acid Ther 28 10-22

[9] Swayze EE(2017)The effects of 2'-O-methoxyethyl containing antisense oligonucleotides on platelets in human clinical trials Nucleic Acid Ther 27 121-129

[10] Benson MD(2019)An integrated safety analysis of infants and children with symptomatic spinal muscular atrophy (SMA) treated with nusinersen in seven clinical trials CNS Drugs 33 919-932

[1] Alves CRR(2020)Serum creatinine is a biomarker of progressive denervation in spinal muscular atrophy Neurology 94 e921-e931

[2] Zhang R(2010)RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform Annu Rev Pharmacol Toxicol 50 259-293

[3] Johnstone AJ(2018)Inotersen treatment for patients with hereditary transthyretin amyloidosis N Engl J Med 379 22-31

[4] Garner R(2019)Adult CSF total protein: higher upper reference limits should be considered worldwide. A web-based survey J Neurol Sci 396 48-51

[5] Nwe PH(2019)Adult CSF total protein upper reference limits should be age-partitioned and significantly higher than 0.45 g/L: a systematic review J Neurol 266 616-624

[6] Siranosian JJ(2006)Antisense oligonucleotides: from design to therapeutic application Clin Exp Pharmacol Physiol 33 533-540

[7] Swoboda KJ(2016)Integrated safety assessment of 2'-O-methoxyethyl chimeric antisense oligonucleotides in nonhuman primates and healthy human volunteers Mol Ther 24 1771-1782

[8] Bennett CF(2018)The effects of 2'-O-methoxyethyl oligonucleotides on renal function in humans Nucleic Acid Ther 28 10-22

[9] Swayze EE(2017)The effects of 2'-O-methoxyethyl containing antisense oligonucleotides on platelets in human clinical trials Nucleic Acid Ther 27 121-129

[10] Benson MD(2019)An integrated safety analysis of infants and children with symptomatic spinal muscular atrophy (SMA) treated with nusinersen in seven clinical trials CNS Drugs 33 919-932