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Clinical Trial
. 2025 Feb 3;8(2):e2459058.
doi: 10.1001/jamanetworkopen.2024.59058.

Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial

Sabrina Paganoni  1   2 , Christina N Fournier  3 , Eric A Macklin  1   4 , Lori B Chibnik  1   4   5 , Melanie Quintana  6 , Benjamin R Saville  6 , Michelle A Detry  6 , Matteo Vestrucci  6 , Joe Marion  6 , Anna McGlothlin  6 , Senda Ajroud-Driss  7 , Marianne Chase  1 , Lindsay Pothier  1 , Brittney A Harkey  1 , Hong Yu  1 , Alexander V Sherman  1 , Jeremy M Shefner  8 , Meghan Hall  8 , Gale Kittle  8 , James D Berry  1 , Suma Babu  1 , Jinsy Andrews  9 , Derek Dagostino  1 , Eric Tustison  1 , Elisa Giacomelli  1 , Erica Scirocco  1 , Gustavo Alameda  10 , Eduardo Locatelli  10   11 , Doreen Ho  1 , Adam Quick  12 , Jonathan Katz  13 , Daragh Heitzman  14 , Stanley H Appel  15 , Sheetal Shroff  15 , Kevin Felice  16 , Nicholas J Maragakis  17 , Zachary Simmons  18 , Timothy M Miller  19 , Nicholas Olney  20 , Michael D Weiss  21 , Stephen A Goutman  22 , Joseph Americo Fernandes  23 , Omar Jawdat  24 , Margaret Ayo Owegi  25 , Laura A Foster  26 , Tuan Vu  27 , Hristelina Ilieva  28 , Daniel S Newman  29 , Ximena Arcila-Londono  29 , Carlayne E Jackson  30 , Shafeeq Ladha  8 , Terry Heiman-Patterson  31 , James B Caress  32 , Andrea Swenson  33 , Amanda Peltier  34 , Richard Lewis  35 , Dominic Fee  36 , Matthew Elliott  37 , Richard Bedlack  38 , Edward J Kasarskis  39 , Lauren Elman  40 , Jeffrey Rosenfeld  41 , David Walk  42 , Courtney McIlduff  43 , Paul Twydell  44 , Eufrosina Young  45 , Kristin Johnson  46 , Kourosh Rezania  47 , Namita A Goyal  48 , Jeffrey A Cohen  49 , Michael Benatar  50 , Vovanti Jones  51 , Jonathan Glass  3 , Jaimin Shah  52 , Said R Beydoun  53 , James P Wymer  54 , Lindsay Zilliox  55 , Shakti Nayar  56 , Gary L Pattee  57 , Jennifer Martinez-Thompson  58 , Brittany Harvey  59 , Shital Patel  59 , Paul Mahoney  60 , Petra W Duda  59 , Merit E Cudkowicz  1 ; HEALEY ALS Platform Trial Study Group
Collaborators, Affiliations
Clinical Trial

Efficacy and Safety of Zilucoplan in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial

Sabrina Paganoni et al. JAMA Netw Open. .

Abstract

Importance: The etiology of amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease, is unknown. However, neuroinflammation and complement activation may play a role in disease progression.

Objective: To determine the effects of zilucoplan, an inhibitor of complement C5, in individuals with ALS.

Design, setting, and participants: Zilucoplan was tested as regimen A of the HEALEY ALS Platform Trial, a phase 2 to 3 multicenter, randomized, double-blind, placebo-controlled perpetual platform clinical trial with sharing of trial infrastructure and placebo data across multiple regimens. Regimen A was conducted from August 17, 2020, to May 4, 2022. A total of 162 participants were randomized to receive zilucoplan (122 [75.3%]) or regimen-specific placebo (40 [24.7%]). An additional 124 concurrently randomized participants were randomized to receive placebo in other regimens.

Interventions: Eligible participants were randomized in a 3:1 ratio to receive zilucoplan or matching placebo within strata of edaravone and/or riluzole use for a planned duration of 24 weeks. Active drug (zilucoplan, 0.3 mg/kg) and placebo were provided for daily subcutaneous dosing.

Main outcomes and measures: The primary end point was change in disease severity from baseline through 24 weeks as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival, analyzed using a bayesian shared-parameter model and reported as disease rate ratio (DRR; <1 indicating treatment benefit). The study included prespecified rules for early stopping for futility. Outcome analyses were performed in the full analysis set comparing the zilucoplan group with the total shared placebo group (n = 164).

Results: Among the 162 participants who were randomized (mean [SD] age, 59.6 [11.3]; 99 [61.1%] male), 115 (71.0%) completed the trial. The estimated DRR common to ALSFRS-R and survival was 1.08 (95% credible interval, 0.87-1.31; posterior probability of superiority, 0.24). The trial was stopped early for futility. No unexpected treatment-related risks were identified.

Conclusions and relevance: In this randomized clinical trial of zilucoplan in ALS, treatment did not alter disease progression. The adaptive platform design of the HEALEY ALS Platform Trial made it possible to test a new investigational product with efficient use of time and resources.

Trial registration: ClinicalTrials.gov Identifier: NCT04297683.

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Conflict of interest statement

Conflict of Interest Disclosures: Dr Paganoni reported receiving grant funding from the AMG Charitable Foundation, Tackle ALS, the ALS Association, ALS Finding a Cure, Muscular Dystrophy Association (MDA), ALS ONE, Arthur M. Blank Family Foundation, I AM ALS, Tambourine ALS Collaborative Cohort, UCB, Biohaven Ltd, Clene Inc, and Prilenia Therapeutics during the conduct of the study and consulting for Amylyx Pharmaceuticals Inc, Revalesio, BMS, Clene Inc, Prilenia Therapeutics, Eikonizo Therapeutics, SOLA Pharmaceuticals, and PharmAust, grant funding from Amylyx Pharmaceuticals Inc, Eledon Pharmaceuticals Inc, Alector Inc, Seelos Therapeutics Inc, Calico Life Sciences LLC, Denali Therapeutics Inc, the National Institutes of Health (NIH), the Centers for Disease Control and Prevention, and the Department of Defense, personal fees from Arrowhead Pharmaceuticals, Biogen Inc, Johnson & Johnson, Merck and Co Inc, and Cytokinetics Incorporated, and speaker fees for educational courses from PeerView, Medscape, and i3 Health outside the submitted work. Dr Macklin reported receiving grant funding from UCB during the conduct of the study and grant funding paid to institution from AI Therapeutics, Alector Inc, Biohaven Ltd, Calico Life Sciences LLC, Clene Inc, Denali Therapeutics Inc, ITB-MED, Janssen Global Services LLC, Eli Lilly and Company, Mitubishi Tanabe Pharma America Inc, Neurizon Therapeutics, Prilenia Therapeutics, Revalesio, Seelos Therapeutics Inc, UCB–Ra Pharmaceuticals, and Woolsey Pharmaceuticals Inc, serving on advisory boards for Annexon Biosciences, Bial, Cortexyme, Chase Therapeutics, Enterin, Hillhurst Bio, Merck and Co Inc, nQ Medical Inc, and Partner Therapeutics Inc, and serving on steering committees for Biogen Inc, Stoparkinson Healthcare, and UCB, and serving on data safety monitoring boards for argenx, NeuroSense, Novartis AG, and Sanofi SA outside the submitted work. Dr Saville reported owning and receiving personal fees from Adaptix Trials LLC outside the submitted work. Dr Detry reported consulting for Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital (MGH) with fees paid to employer during the conduct of the study. Dr Vestrucci reported consulting for Sean M Healey & AMG Center for ALS at MGH with fees paid to employer during the conduct of the study. Dr Marion reported receiving personal fees from Berry Consultants during the conduct of the study. Dr McGlothlin reported consulting for Sean M Healey & AMG Center for ALS at MGH with fees paid to employer during the conduct of the study. Dr Ajroud-Driss reported serving on the advisory board of Amylyx Pharmaceuticals Inc and receiving speaking fees from Biogen Inc during the conduct of the study. Dr Chase reported receiving grant funding from UCB, Biohaven Ltd, Clene Inc, and Prilenia Therapeutics during the conduct of the study. Dr Sherman reported receiving grant funding from UCB during the conduct of the study and receiving grant funding from the US Food and Drug Administration, the National Institute of Neurological Disorders and Stroke (NINDS) of the NIH, Biogen Inc, Mitsubishi Tanabe Pharma America, Amylyx Pharmaceuticals Inc, and the ALS Association outside the submitted work. Dr Shefner reported personal fees from Cytokinetics Incorporated, Amylyx Pharmaceuticals Inc, PTC Therapeutics, Sanofi SA, Mitsubishi Tanabe Pharma America, Vertex Pharmaceuticals Incorporated, Novartis AG, NeuroSense Therapeutics Ltd, and from AcuraStem and grant funding from Cytokinetics Incorporated, Amylyx Pharmaceuticals Inc, QurAlis, Sanofi SA, Mitsubishi Tanabe Pharma America, SwanBio Therapeutics Inc, and NeuroSense Therapeutics Ltd outside the submitted work. Dr Berry reported receiving grant funding from Amylyx Pharmaceuticals Inc, Alexion Pharmaceuticals Inc, Biogen Inc, Brainstorm Cell Therapeutics Inc, Mitsubishi Tanabe Pharma America Inc, ALS Association, ALS One, Tambourine ALS Collaborative Cohort, Hop On a Cure, NINDS, and the Department of Defense and personal fees from Amylyx Pharmaceuticals Inc, Alexion Pharmaceuticals Inc, Mitsubishi Tanabe Pharma America Inc, Janssen Global Services LLC, Rapa Therapeutics, MDA, PeerView, and Projects in Knowledge outside the submitted work. Dr Babu reported receiving grant funding from Biogen Inc, Ionis Pharmaceuticals Inc, uniQure NQ, OrphAI Therapeutics, MarvelBiome Inc, and Novartis AG outside the submitted work. Dr Andrews reported serving on the data safety monitoring boards of AL-S Pharma AG, QurAlis, and Sanofi SA, receiving personal fees from Biogen Inc, Cytokinetics Incorporated, Amylyx Pharmaceuticals Inc, Apellis Pharmaceuticals, Wave Life Sciences Ltd, and Revalesio, and receiving grant funding to institution from Amylyx Pharmaceuticals Inc, Biogen Inc, grant funding from Cytokinetics Incorporated, and the HEALEY ALS Platform Trial (Ra–Biohaven Ltd–Clene Inc–Prilenia Therapeutics–Calico Life Sciences LLC) outside the submitted work. Dr Giacomelli reported receiving grant funding from the AMG Charitable Foundation, Tackle ALS, ALS Association, ALS Finding A Cure, MDA, ALS ONE, Arthur M. Blank Family Foundation, I AM ALS, and Tambourine ALS Collaborative Cohort during the conduct of the study. Dr Scirocco reported receiving grant funding from the AMG Charitable Foundation, Tackle ALS, ALS Association, ALS Finding A Cure, MDA, ALS One, Arthur M. Blank Family Foundation, I AM ALS, and Tambourine ALS Collaborative Cohort during the conduct of the study. Dr Ho reported receiving research funding from Prilenia Therapeutics, Ra Pharmaceuticals, Biohaven Ltd, Clene Inc, Seelos Therapeutics Inc, Calico Life Sciences LLC, Denali Therapeutics Inc, Biogen Inc, Transposon Therapeutics Inc, Sanofi SA, the Neurodegenerative Alzheimer’s Disease and Amyotrophic Lateral Sclerosis Basket Trial, and Genentech Inc and serving on the advisory boards of Biogen Inc, Sanofi SA, and Alexion Pharmaceuticals Inc outside the submitted work. Dr Heitzman reported receiving grant funding from the MDA, ALS Association, and NINDS during the conduct of the study and receiving study support from Abcuro Inc, AbbVie Inc, AbbVie Inc–Calico Life Sciences LLC, Biohaven Ltd, Cytokinetics Incorporated, Denali Therapeutics Inc, Immunovant Inc, Mitsubishi Tanebe Pharma America, Prelenia Therapeutics, Ra, Amylyx Pharmaceuticals Inc, and Trehelose and personal fees from Amylyx Pharmaceuticals Inc outside the submitted work. Dr Appel reported serving as chair of the scientific advisory board for Coya Therapeutics Inc, receiving grant funding to institution from Coya Therapeutics Inc, consulting for Eledon Pharmaceuticals Inc, and serving as scientific advisory for Mitsubishi Tanebe Pharma America during and outside the submitted work. Dr Shroff reported receiving speaking fees from UCB, Alnylam Pharmaceuticals Inc, AstraZeneca, and argenx and serving on the advisory boards for AstraZeneca and argenx outside the submitted work. Dr Maragakis reported receiving grant funding from the NINDS of the NIH and the Department of Defense–Amyotrophic Lateral Sclerosis Research Program during the conduct of the study and personal fees from Nura Bio, Novartis AG, and AKAVA Therapeutics Inc and nonfinancial support from Janssen Global Services LLC and Secretome Therapeutics outside the submitted work. Dr Simmons reported receiving grant funding from MGH during the conduct of the study and personal fees from Amylyx Pharmaceuticals Inc, Biogen Inc, and Corcept Therapeutics Inc, and grant funding from Clene Inc, Sanofi SA, and MGH outside the submitted work. Dr Miller reported receiving resources to complete the study from Healey Center at MGH through HEALEY ALS Platform Trial during the conduct of the study and consulting for Ionis Pharmaceuticals Inc, Biogen Inc, and Arbor Biotechnologies, licensing agreements from Ionis Pharmaceuticals Inc and C2N Diagnostics, and honorarium from Denali Therapeutics Inc outside the submitted work. Dr Olney reported receiving funding to conduct and run the trial from the HEALEY ALS Platform Trial during the conduct of the study. Dr Weiss reported receiving personal fees from UCB for helping with a webinar for their myasthenia gravis indication for zilucoplan, serving on the advisory boards of Cytokinetics Incorporated, Mitsubishi Tanebe Pharma America, Amylyx Pharmaceuticals Inc, and Biogen Inc and for serving on the data safety monitoring board of Sanofi SA outside the submitted work. Dr Goutman reported receiving grant funding paid to institution from Healey Center at MGH during the conduct of the study. Dr Fernandes reported receiving grant funding from MGH philanthropy (Clene Inc, Seelos Therapeutics Inc, UCB, Biohaven Ltd, Prilenia Therapeutics, Denali Therapeutics Inc, and Calico Life Sciences LLC) during the conduct of the study and receiving personal fees for serving as a medical monitor from Columbia University (Tsumura & Co) and grant funding for serving as site principal investigator from Columbia University, PTC Therapeutics, Clene Inc, and MGH philanthropy (NIH) outside the submitted work. Dr Foster reported receiving grant funding from the Sean M. Healey & AMG Center for ALS during the conduct of the study. Dr Vu reported receiving reimbursement for trial activities from the Department of Neurology, University of South Florida, during the conduct of the study and receiving personal fees for serving on speaking bureaus and for consulting work unrelated to ALS and the HEALEY trials outside the submitted work. Dr Ilieva reported receiving grant funding from ALS Association and Akcea Therapeutics Inc and philanthropic support from Vickie and Jack Farber for the Weinberg ALS Center during the conduct of the study. Dr Jackson reported receiving grant funding from MGH during the conduct of the study. Dr Ladha reported receiving personal fees from Biogen Inc, Amylyx Pharmaceuticals Inc, Sanofi SA, and Genentech outside the submitted work. Dr Heiman-Patterson reported receiving grant funding from the HEALEY ALS Platform Trial during the conduct of the study and receiving grant funding from MT Pharma, Amylyx Pharmaceuticals Inc, and ALS Association and serving on the medical advisory board for MT Pharma, Amylyx Pharmaceuticals Inc, and Novartis AG outside the submitted work. Dr Lewis reported serving on the data safety monitoring board for clinical trials for Novartis AG, Intellia Therapeutics Inc, and Boehringer Ingelheim and consulting for Annexon Biosciences, argenx, Dianthus Therapeutics Inc, Grifols SA, CSL Behring, Nuvig Therapeutics, Sanofi SA, Immunovant Inc, and Johnson & Johnson outside the submitted work. Dr Elliott reported receiving grant funding from UCB during the conduct of the study and receiving grant funding from Denali Therapeutics Inc, Calico Life Sciences LLC, ALS Association, Prilenia Therapeutics, Clene Inc, Biohaven Ltd, and MediciNova Inc outside the submitted work. Dr Bedlack reported receiving grant funding from Duke University during the conduct of the study. Dr Rosenfeld reported receiving grant funding from Healy Center MGH during the conduct of the study. Dr Walk reported receiving personal fees from Mitsubishi Tanabe Pharma America, Amylyx Pharmaceuticals Inc, and Clene Inc outside the submitted work. Dr McIlduff reported support from the Master Site Clinical Trial Agreement at Sean M. Healey and AMG Center for ALS at MGH during the conduct of the study. Dr Johnson reported receiving grant funding from MGH during the conduct of the study and receiving speaking fees from argenx outside the submitted work. Dr Goyal reported receiving grant funding from the HEALEY ALS Platform Trial during the conduct of the study and receiving grant funding from Abcuro Inc, Amylyx Pharmaceuticals Inc, Annexon Biosciences, Calico Life Sciences LLC, Clene Inc, Cytokinetics Incorporated, Fulcrum Therapeutics Inc, Janssen Global Services LLC, Kezar Life Sciences Inc, MediciNova Inc, MT Pharma, PepGen Inc, PTC Therapeutics, F. Hoffmann–La Roche AG, Sanofi SA, and Transposon Therapeutics Inc and serving on the speakers’ bureau for argenx and CSL Behring outside the submitted work. Dr Benatar reported per patient reimbursement from MGH during the conduct of the study and consulting for Prilenia Therapeutics, Alector, Biogen Inc, Novartis AG, BMS, Woolsey, Eli Lilly and Company, uniQure NQ, and Arrowhead Pharmaceuticals outside the submitted work. Dr Shah reported receiving grant funding from MDA, argenx, and Abcuro Inc outside the submitted work. Dr Beydoun reported receiving grant funding from University of Southern California during the conduct of the study and receiving grant funding from Abcuro Inc, Janssen Global Services LLC, Regeneron Pharmaceuticals Inc, Sanofi SA and personal fees from Alexion Pharmaceuticals Inc, Alnylam Pharmaceuticals Inc, argenx, AstraZeneca, CSL Behring, Takeda Pharmaceutical Company Limited, and UCB outside the submitted work. Dr Wymer reported receiving grant funding from MGH during the conduct of the study. Dr Harvey reported receiving personal fees from UCB during the conduct of the study and outside the submitted work. Dr Duda reported holding stock and stock options from UCB outside the submitted work. Dr Cudkowicz reported receiving grant funding for the trial from UCB during the conduct of the study. No other disclosures were reported.

Figures

Figure 1.
Figure 1.. Study Flow Diagram
Participants could have multiple reasons for exclusion from the master protocol and could screen for the master protocol more than once. The most common reasons for exclusion were not meeting the criterion for slow vital capacity of at least 50% (66 of 143 [46% of person-visits]), having a clinically significant unstable medical condition (other than amyotrophic lateral sclerosis [ALS]) that would pose a risk to the participant (44 of 143 [31% of person-visits]), and having used investigational treatments for ALS within 5 half-lives (if known) or 30 days (whichever is longer) prior to the master protocol screening visit (24 of 143 [17% of person-visits]). The 29 participants who completed the regimen in the regimen-specific placebo are included in the 137 shared placebo who completed the study.
Figure 2.
Figure 2.. Change in Disease Severity Over 24 Weeks as Measured by Amyotrophic Lateral Sclerosis Functional Rating Scale–Revised (ALSFRS-R) and Survival (Primary Outcome)
A, Boxplots of the ALSFRS-R change from baseline over time for the active treatment (light blue), the regimen placebo (light tan), and the shared placebo (dark tan). The bottom of each box represents the 25th percentile of the ALSFRS-R change from baseline values and the top of the box represents the 75th percentile. The horizontal line within each box represents the median value. The whiskers extend to the most extreme data point that is no further than 1.5 times the length of the box away from the box, with outlier values beyond this range shown by open circles. Solid points are the raw mean values at each visit, with different shapes and colors representing active treatment (light blue circles), the regimen placebo group (light tan squares), and the shared placebo group (dark tan triangles). The solid diagonal lines are the model-estimated change in ALSFRS-R over time, adjusting for covariates. The estimate for the regimen placebo group shares information from the placebo groups in other regimens. The numbers below the panel show the number of participants with known ALSFRS-R outcomes at each visit. The summary excludes all ALSFRS-R data from participants who died or had a death-equivalent event (permanent assisted ventilation [PAV]). ALSFRS-R scores range from 11.0 to 47.0, with higher scores indicating better function. B, Kaplan-Meier curves for death or PAV per arm and the model-estimated exponential curves for the active treatment and pooled placebo groups. The numbers below the panel indicate the number of participants exposed and the number of death or PAV events per arm.
Figure 3.
Figure 3.. Study Timeline
A, The actual timeline for trial completion after the futility criterion was met at the February 2022 interim analysis. B, The projected timeline for trial completion if interim analyses did not occur and all participants completed the full 24 weeks of the randomized placebo-controlled portion of the regimen. Using the projected dates of trial completion, the sum of all expected visits that would have occurred between the actual and projected last participant last visit (LPLV) was calculated using individual participant visit schedules generated from the electronic data capture system. FPFV indicates first participant first visit; OLE, open-label extension; RCT, randomized clinical trial; and TLR, top line results. aPer the regimen specific appendix, the final study visit for a participant occurred 40 plus or minus 3 days after discontinuing investigational product treatment.
See this image and copyright information in PMC

Comment in

  • Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial.
    Writing Committee for the HEALEY ALS Platform Trial; Andrews J, Paganoni S, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Young E, Chase M, Pothier L, Harkey B, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Connolly MR, Berry JD, D'Agostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Heitzman D, Ajroud-Driss S, Appel SH, Shroff S, Katz J, Felice K, Maragakis NJ, Simmons Z, Goutman SA, Olney N, Miller T, Fernandes JA, Ilieva H, Jawdat O, Weiss MD, Foster L, Vu T, Ladha S, Owegi MA, Newman DS, Arcila-Londono X, Jackson CE, Swenson A, Heiman-Patterson T, Caress J, Fee D, Peltier A, Lewis R, Rosenfeld J, Walk D, Johnson K, Elliott M, Kasarskis EJ, Rutkove S, McIlduff CE, Bedlack R, Elman L, Goyal NA, Rezania K, Twydell P, Benatar M, Glass J, Cohen JA, Jones V, Zilliox L, Wymer JP, Beydoun SR, Shah J, Pattee GL, Martinez-Thompson J, Nayar S, Granit V, Donohue M, Grossman K, Campbell DJ, Qureshi IA, Cudkowicz ME, Babu S. Writing Committee for the HEALEY ALS Platform Trial, et al. JAMA Neurol. 2025 Apr;82(4):333-343. doi: 10.1001/jamaneurol.2024.5249. Epub 2025 Feb 17. JAMA Neurol. 2025. PMID: 40067754 Free PMC article. Clinical Trial.

References

    1. Appel SH, Beers DR, Zhao W. Amyotrophic lateral sclerosis is a systemic disease: peripheral contributions to inflammation-mediated neurodegeneration. Curr Opin Neurol. 2021;34(5):765-772. doi: 10.1097/WCO.0000000000000983 - DOI - PubMed
    1. Kjældgaard AL, Pilely K, Olsen KS, et al. Complement profiles in patients with amyotrophic lateral sclerosis: a prospective observational cohort study. J Inflamm Res. 2021;14:1043-1053. doi: 10.2147/JIR.S298307 - DOI - PMC - PubMed
    1. Apostolski S, Nikolić J, Bugarski-Prokopljević C, Miletić V, Pavlović S, Filipović S. Serum and CSF immunological findings in ALS. Acta Neurol Scand. 1991;83(2):96-98. doi: 10.1111/j.1600-0404.1991.tb04656.x - DOI - PubMed
    1. Mantovani S, Gordon R, Macmaw JK, et al. Elevation of the terminal complement activation products C5a and C5b-9 in ALS patient blood. J Neuroimmunol. 2014;276(1-2):213-218. doi: 10.1016/j.jneuroim.2014.09.005 - DOI - PubMed
    1. Lee JD, Kumar V, Fung JN, Ruitenberg MJ, Noakes PG, Woodruff TM. Pharmacological inhibition of complement C5a-C5a1 receptor signalling ameliorates disease pathology in the hSOD1G93A mouse model of amyotrophic lateral sclerosis. Br J Pharmacol. 2017;174(8):689-699. doi: 10.1111/bph.13730 - DOI - PMC - PubMed

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