References
- Kiernan MC, Vucic S, Talbot K, et al. Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nat Rev Neurol. 2020 Dec 18;17:1–15.
- Kiernan MC, Vucic S, Cheah BC, et al. Amyotrophic lateral sclerosis. Lancet. 2011 Mar 12;377(9769):942–955. doi: 10.1016/S0140-6736(10)61156-7
- Lacomblez L, Bensimon G, Leigh PN, et al. Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic lateral sclerosis/riluzole study group II. The Lancet. 1996;347(9013):1425–1431. doi: 10.1016/S0140-6736(96)91680-3
- 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):585–591. doi: 10.1056/NEJM199403033300901
- Writing Group EM-ASG. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017 Jul;16(7):505–512. doi: 10.1016/S1474-4422(17)30115-1
- Paganoni S, Macklin EA, Hendrix S, et al. Trial of sodium phenylbutyrate-taurursodiol for amyotrophic lateral sclerosis. N Engl J Med. 2020 Sep 3;383(10):919–930. doi: 10.1056/NEJMoa1916945
- Miller T, Cudkowicz M, Shaw PJ, et al. Phase 1-2 trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2020 Jul 9;383(2):109–119. doi: 10.1056/NEJMoa2003715
- Witzel S, Maier A, Steinbach R, et al. Safety and effectiveness of long-term intravenous administration of edaravone for treatment of patients with amyotrophic lateral sclerosis. JAMA Neurol. 2022 Feb 1;79(2):121–130. doi: 10.1001/jamaneurol.2021.4893
- Vucic S, Kiernan MC. Transcranial magnetic stimulation for the assessment of neurodegenerative disease. Neurotherapeutics. 2017 Jan;14(1):91–106. doi: 10.1007/s13311-016-0487-6
- Burg T, Van Den Bosch L. Abnormal energy metabolism in ALS: a key player? Curr Opin Neurol. 2023 Aug 1;36(4):338–345.
- Hor JH, Santosa MM, Lim VJW, et al. ALS motor neurons exhibit hallmark metabolic defects that are rescued by SIRT3 activation. Cell Death Differ. 2021 Apr;28(4):1379–1397. doi: 10.1038/s41418-020-00664-0
- Robinson AP, Zhang JZ, Titus HE, et al. Nanocatalytic activity of clean-surfaced, faceted nanocrystalline gold enhances remyelination in animal models of multiple sclerosis. Sci Rep. 2020 Feb 11;10(1):1936. doi: 10.1038/s41598-020-58709-w
- Cantó C, Menzies KJ, Auwerx J. NAD(+) metabolism and the control of energy homeostasis: a balancing act between mitochondria and the nucleus. Cell Metab. 2015 Jul 7;22(1):31–53.
- Vucic S, Menon P, Huynh W, et al. Efficacy and safety of CNM-Au8 in amyotrophic lateral sclerosis (RESCUE-ALS study): a phase 2, randomised, double-blind, placebo-controlled trial and open label extension. EClinicalMedicine. 2023 Jun;60:102036. doi: 10.1016/j.eclinm.2023.102036
- Paganoni S, Berry JD, Quintana M, et al. Adaptive platform trials to transform amyotrophic lateral sclerosis therapy development. Ann Neurol. 2022 Feb;91(2):165–175. doi: 10.1002/ana.26285
- Berry JD, Healey MEC & AMG center announces top line results in ALS platform trial with CMN-Au8. cited 2022 Oct 3. https://wwwmassgeneralorg/news/press-release/healey-amg-center-announces-top-line-results-in-als-platform-trial-with-cmn-au-eight.
- Benatar M, Wuu J, Turner MR. Neurofilament light chain in drug development for amyotrophic lateral sclerosis: a critical appraisal. Brain. 2023 Jul 3;146(7):2711–2716.