Masrori P, Van Damme P. Amyotrophic lateral sclerosis: a clinical review. Eur J Neurol. 2020;27(10):1918–29.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Stetkarova I, Ehler E. Diagnostics of amyotrophic lateral sclerosis: up to date. Diagnostics (Basel). 2021;11(2).

Kiernan MC, et al. Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nat Rev Neurol. 2021;17(2):104–18.

Article  PubMed  Google Scholar 

Dorst J, Ludolph AC, Huebers A. Disease-modifying and symptomatic treatment of amyotrophic lateral sclerosis. Ther Adv Neurol Disord. 2018;11:1756285617734734.

Article  PubMed  Google Scholar 

Miller RG, Mitchell JD, Moore DH. Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND). Cochrane Database Syst Rev. 2012;2012(3):CD001447.

Bensimon G, Lacomblez L, Meininger V. A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group. N Engl J Med, 1994;330(9):585–91.

Lacomblez L, et al. Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis/Riluzole Study Group II. Lancet. 1996;347(9013):1425–31.

Bensimon G, et al. A study of riluzole in the treatment of advanced stage or elderly patients with amyotrophic lateral sclerosis. J Neurol. 2002;249(5):609–15.

Article  CAS  PubMed  Google Scholar 

Yanagisawa N, et al. Efficacy and safety of riluzole in patients with amyotrophic lateral sclerosis: double-blind placebo-controlled study in Japan. Igakuno Ayumi. 1997;182(11):851–66.

Google Scholar 

Andrews JA, et al. Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2020;21(7–8):509–18.

Article  CAS  PubMed  Google Scholar 

Lacomblez L, et al. Long-term safety of riluzole in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord. 2002;3(1):23–9.

Article  CAS  PubMed  Google Scholar 

Cruz MP. Edaravone (Radicava): a novel neuroprotective agent for the treatment of amyotrophic lateral sclerosis. P t. 2018;43(1):25–8.

PubMed  PubMed Central  Google Scholar 

Abe K, et al. Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients. Amyotroph Lateral Scler Frontotemporal Degener. 2014;15(7–8):610–7.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Edaravone Als 16 Study, G., A post-hoc subgroup analysis of outcomes in the first phase III clinical study of edaravone (MCI-186) in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2017;18(sup1):11–19.

Tanaka M, et al. A 24-week, phase III, double-blind, parallel-group study of Edaravone (MCI-186) for treatment of amyotrophic lateral sclerosis (ALS) (P3.189). Neurology. 2016;86(16 Supplement): p. P3.189.

Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017;16(7):505–512.

Exploratory double-blind, parallel-group, placebo-controlled study of edaravone (MCI-186) in amyotrophic lateral sclerosis (Japan ALS severity classification: Grade 3, requiring assistance for eating, excretion or ambulation). Amyotroph Lateral Scler Frontotemporal Degener. 2017;18(sup1):40–48.

Witzel S, et al. Safety and effectiveness of long-term intravenous administration of edaravone for treatment of patients with amyotrophic lateral sclerosis. JAMA Neurol. 2022;79(2):121–30.

Article  PubMed  Google Scholar 

Ishizaki K, et al. Real-world safety of the novel, free radical scavenger edaravone for amyotrophic lateral sclerosis patients: data from the post-marketing surveillance SUNRISE Japan. Neurol Clin Neurosci. 2021;9(3):223–9.

Article  CAS  Google Scholar 

Paganoni S, et al. Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis. Muscle Nerve. 2021;63(1):31–9.

Article  CAS  PubMed  Google Scholar 

Paganoni S, et al. Trial of sodium phenylbutyrate–taurursodiol for amyotrophic lateral sclerosis. N Engl J Med. 2020;383(10):919–30.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Paganoni S, et al. Survival analyses from the CENTAUR trial in amyotrophic lateral sclerosis: evaluating the impact of treatment crossover on outcomes. Muscle Nerve. 2022;66(2):136–41.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Inc AP. Phase III trial of AMX0035 for amyotrophic lateral sclerosis treatment. 2021. https://ClinicalTrials.gov/show/NCT05021536.

Van Mossevelde S, et al. Relationship between C9orf72 repeat size and clinical phenotype. Curr Opin Genet Dev. 2017;44:117–24.

Article  PubMed  Google Scholar 

AI Therapeutics I. Study of safety, tolerability, and biological activity of LAM-002A in C9ORF72-associated amyotrophic lateral sclerosis. 2021. https://ClinicalTrials.gov/show/NCT05163886.

Inc A. A phase 2 study to evaluate AL001 in C9orf72-associated ALS. 2021. https://ClinicalTrials.gov/show/NCT05053035.

Ltd., W.L.S., Study of WVE-004 in patients with C9orf72-associated amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). 2021. https://ClinicalTrials.gov/show/NCT04931862.

Florida UO. Safety and therapeutic potential of the FDA-approved drug metformin for C9orf72 ALS/FTD. 2020. https://ClinicalTrials.gov/show/NCT04220021.

Biogen, A study to assess the safety, tolerability, and pharmacokinetics of BIIB078 in adults with C9ORF72-associated amyotrophic lateral sclerosis. 2018. https://ClinicalTrials.gov/show/NCT03626012.

Transposon Therapeutics I. A phase 2a study of TPN-101 in patients with C9ORF72 ALS/FTD. 2021. https://ClinicalTrials.gov/show/NCT04993755.

Peggion C, et al. SOD1 in ALS: taking stock in pathogenic mechanisms and the role of glial and muscle cells. Antioxidants (Basel). 2022;11(4).

Miller TM, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099–110.

Article  CAS  PubMed  Google Scholar 

Biogen, A Study of BIIB067 When Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation. 2021. https://ClinicalTrials.gov/show/NCT04856982.

Nolan M, Talbot K, Ansorge O. Pathogenesis of FUS-associated ALS and FTD: insights from rodent models. Acta Neuropathol Commun. 2016;4(1):99.

Article  PubMed  PubMed Central  Google Scholar 

Korobeynikov VA, et al. Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nat Med. 2022;28(1):104–16.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Ionis Pharmaceuticals I. A study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ION363 in amyotrophic lateral sclerosis participants with fused in sarcoma mutations (FUS-ALS). 2021. https://ClinicalTrials.gov/show/NCT04768972.

Elden AC, et al. Ataxin-2 intermediate-length polyglutamine expansions are associated with increased risk for ALS. Nature. 2010;466(7310):1069–75.

Article  CAS  PubMed  PubMed Central  Google Scholar 

van den Heuvel DM, et al. Taking a risk: a therapeutic focus on ataxin-2 in amyotrophic lateral sclerosis? Trends Mol Med. 2014;20(1):25–35.

Article  PubMed  Google Scholar 

Biogen. A study to assess the safety, tolerability, and effect on disease progression of BIIB105 in participants with amyotrophic lateral sclerosis (ALS) and participants with the ALS Ataxin-2 (ATXN2) Genetic Mutation (ALSpire). 2020.

Pinto AC, et al. Respiratory assistance with a non-invasive ventilator (Bipap) in MND/ALS patients: survival rates in a controlled trial. J Neurol Sci. 1995;129(Suppl):19–26.

Article  PubMed  Google Scholar 

Kleopa KA, et al. Bipap improves survival and rate of pulmonary function decline in patients with ALS. J Neurol Sci. 1999;164(1):82–8.

Article  CAS  PubMed  Google Scholar 

Bourke SC, et al. Effects of non-invasive ventilation on survival and quality of life in patients with amyotrophic lateral sclerosis: a randomised controlled trial. Lancet Neurol. 2006;5(2):140–7.

Article  PubMed  Google Scholar 

Vitacca M, et al. Impact of an early respiratory care programme with non-invasive ventilation adaptation in patients with amyotrophic lateral sclerosis. Eur J Neurol. 2018;25(3):556-e33.

Article  CAS  PubMed  Google Scholar 

Dorst J, Ludolph AC. Non-invasive ventilation in amyotrophic lateral sclerosis. Ther Adv Neurol Disord. 2019;12:1756286419857040.

Article  PubMed  PubMed Central  Google Scholar 

Weiss MD, et al. A randomized trial of mexiletine in ALS: safety and effects on muscle cramps and progression. Neurology. 2016;86(16):1474–81.

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cruz MP. Nuedexta for the treatment of pseudobulbar affect: a condition of involuntary crying or laughing. P T. 2013;38(6):325–8.

PubMed  PubMed Central  Google Scholar 

Panitch HS, et al. Randomized, controlled trial of dextromethorphan/quinidine for pseudobulbar affect in multiple sclerosis. Ann Neurol. 2006;59(5):780–7.

Article  CAS  PubMed  Google Scholar 

Brooks BR, et al. Treatment of pseudobulbar affect in ALS with dextromethorphan/quinidine: a randomized trial. Neurology. 2004;63(8):1364–70.

Article  CAS  PubMed  Google Scholar 

Pioro EP, et al. Dextromethorphan plus ultra low-dose quinidine reduces pseudobulbar affect. Ann Neurol. 2010;68(5):693–702.

Article  CAS  PubMed  Google Scholar 

Smith R, et al. Enhanced bulbar function in amyotrophic lateral sclerosis: the nuedexta treatment trial. Neurotherapeutics. 2017;14(3):762–72.

Article  CAS  PubMed  PubMed Central  Google Scholar