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Welcome to the
Pooled Resource Open-Access ALS Clinical Trials Database
About Us

About PRO-ACT

The PRO-ACT (Pooled Resource Open-access ALS Clinical Trials) platform houses the largest ALS clinical trials dataset ever created. PRO-ACT contains 11675 ALS patient records from multiple completed clinical trials. The PRO-ACT initiative merges data from existing publicly- and privately-conducted ALS clinical trials to generate an invaluable resource for accelerating discovery in the field of ALS.

Data within PRO-ACT was graciously donated by the following organizations:
Amylyx Pharmaceuticals, Inc.
Cytokinetics, Inc.
Knopp Biosciences
Neuraltus Pharmaceuticals, Inc.
Northeast ALS Consortium
Novartis
Regeneron Pharmaceuticals, Inc
Sanofi
Teva Pharmaceutical Industries, Ltd

The non-profit organization, Prize4Life, created the PRO-ACT database in partnership with the Northeast ALS Consortium and the Neurological Clinical Research Institute at Massachusetts General Hospital, and with funding from the ALS Therapy Alliance. PRO-ACT was created to provide anyone interested in moving ALS research forward quickly with a powerful tool, thus furthering our shared mission of accelerating the discovery of treatments and a cure for ALS. We thank everyone who has contributed to creation of PRO-ACT including the companies, the researchers, and most of all the ALS patients, past and present, around the world to whom the PRO-ACT initiative is dedicated.

About Neurological Clinical Research Institute

The Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital accelerates translational research in neurological disorders through initiating and testing novel therapies. The NCRI has an extensive history in leading clinical research to find new treatments for neurological diseases including Amyotrophic Lateral Sclerosis (ALS), myasthenia gravis, diabetic neuropathy, stroke, multiple sclerosis, Parkinson’s disease, and Huntington’s disease. For more information, visit www.NCRInstitute.org or www.data4cures.org.

Publications

2014

Gomeni R, Fava M; Pooled Resource Open-Access ALS Clinical Trials Consortium. Amyotrophic lateral sclerosis disease progression model. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Mar;15(1-2):119-29. doi: 10.3109/21678421.2013.838970. Epub 2013 Sep 26. PMID: 24070404.

Atassi N, Berry J, Shui A, Zach N, Sherman A, Sinani E, Walker J, Katsovskiy I, Schoenfeld D, Cudkowicz M, Leitner M. The PRO-ACT database: design, initial analyses, and predictive features. Neurology. 2014 Nov 4;83(19):1719-25. doi: 10.1212/WNL.0000000000000951. Epub 2014 Oct 8. PMID: 25298304; PMCID: PMC4239834.

Hothorn T, Jung HH. RandomForest4Life: a Random Forest for predicting ALS disease progression. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Sep;15(5-6):444-52. doi: 10.3109/21678421.2014.893361. PMID: 25141076.

Ko KD, El-Ghazawi T, Kim D, Morizono H; Pooled Resource Open-Access ALS Clinical Trials Consortium. Predicting the severity of motor neuron disease progression using electronic health record data with a cloud computing Big Data approach. IEEE Symp Comput Intell Bioinforma Comput Biol Proc. 2014 May;2014:14417042. doi: 10.1109/CIBCB.2014.6845506. PMID: 25580472; PMCID: PMC4285703.

2015

Küffner R, Zach N, Norel R, Hawe J, Schoenfeld D, Wang L, Li G, Fang L, Mackey L, Hardiman O, Cudkowicz M, Sherman A, Ertaylan G, Grosse-Wentrup M, Hothorn T, van Ligtenberg J, Macke JH, Meyer T, Schölkopf B, Tran L, Vaughan R, Stolovitzky G, Leitner ML. Crowdsourced analysis of clinical trial data to predict amyotrophic lateral sclerosis progression. Nat Biotechnol. 2015 Jan;33(1):51-7. doi: 10.1038/nbt.3051. Epub 2014 Nov 2. PMID: 25362243.

Zach N, Ennist DL, Taylor AA, Alon H, Sherman A, Kueffner R, Walker J, Sinani E, Katsovskiy I, Cudkowicz M, Leitner ML. Being PRO-ACTive: What can a Clinical Trial Database Reveal About ALS? Neurotherapeutics. 2015 Apr;12(2):417-23. doi: 10.1007/s13311-015-0336-z. PMID: 25613183; PMCID: PMC4404433.

Lunetta C, Lizio A, Melazzini MG, Maestri E, Sansone VA. Amyotrophic Lateral Sclerosis Survival Score (ALS-SS): A simple scoring system for early prediction of patient survival. Amyotroph Lateral Scler Frontotemporal Degener. 2015;17(1-2):93-100. doi: 10.3109/21678421.2015.1083585. Epub 2015 Oct 16. PMID: 26470943.

Tramacere I, Dalla Bella E, Chiò A, Mora G, Filippini G, Lauria G; EPOS Trial Study Group. The MITOS system predicts long-term survival in amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2015 Nov;86(11):1180-5. doi: 10.1136/jnnp-2014-310176. Epub 2015 Apr 17. PMID: 25886781.

2016

Bacci ED, Staniewska D, Coyne KS, Boyer S, White LA, Zach N, Cedarbaum JM; Pooled Resource Open-Access ALS Clinical Trials Consortium. Item response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17(3-4):157-67. doi: 10.3109/21678421.2015.1095930. Epub 2015 Oct 16. PMID: 26473473.

Bedlack RS, Vaughan T, Wicks P, Heywood J, Sinani E, Selsov R, Macklin EA, Schoenfeld D, Cudkowicz M, Sherman A. How common are ALS plateaus and reversals? Neurology. 2016 Mar 1;86(9):808-12. doi: 10.1212/WNL.0000000000002251. Epub 2015 Dec 9. PMID: 26658909; PMCID: PMC4793781.

Proudfoot M, Jones A, Talbot K, Al-Chalabi A, Turner MR. The ALSFRS as an outcome measure in therapeutic trials and its relationship to symptom onset. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Jul-Aug;17(5-6):414-25. doi: 10.3109/21678421.2016.1140786. Epub 2016 Feb 11. PMID: 26864085; PMCID: PMC4950444.

Beaulieu-Jones BK, Greene CS; Pooled Resource Open-Access ALS Clinical Trials Consortium. Semi-supervised learning of the electronic health record for phenotype stratification. J Biomed Inform. 2016 Dec;64:168-178. doi: 10.1016/j.jbi.2016.10.007. Epub 2016 Oct 12. PMID: 27744022.

Taylor AA, Fournier C, Polak M, Wang L, Zach N, Keymer M, Glass JD, Ennist DL; Pooled Resource Open?Access ALS Clinical Trials Consortium. Predicting disease progression in amyotrophic lateral sclerosis. Ann Clin Transl Neurol. 2016 Sep 7;3(11):866-875. doi: 10.1002/acn3.348. PMID: 27844032; PMCID: PMC5099532.

Seibold H, Zeileis A, Hothorn T. Model-Based Recursive Partitioning for Subgroup Analyses. Int J Biostat. 2016 May 1;12(1):45-63. doi: 10.1515/ijb-2015-0032. PMID: 27227717.

2017

Wang J, Luo S. Multidimensional latent trait linear mixed model: an application in clinical studies with multivariate longitudinal outcomes. Stat Med. 2017 Sep 10;36(20):3244-3256. doi: 10.1002/sim.7347. Epub 2017 Jun 1. PMID: 28569393; PMCID: PMC5540878.

Ong ML, Tan PF, Holbrook JD. Predicting functional decline and survival in amyotrophic lateral sclerosis. PLoS One. 2017 Apr 13;12(4):e0174925. doi: 10.1371/journal.pone.0174925. PMID: 28406915; PMCID: PMC5390993.

Huang Z, Zhang H, Boss J, Goutman SA, Mukherjee B, Dinov ID, Guan Y; Pooled Resource Open-Access ALS Clinical Trials Consortium. Complete hazard ranking to analyze right-censored data: An ALS survival study. PLoS Comput Biol. 2017 Dec 18;13(12):e1005887. doi: 10.1371/journal.pcbi.1005887. PMID: 29253881; PMCID: PMC5749893.

Beaulieu-Jones BK, Moore JH. MISSING Data Imputation in the Electronic Health Record Using Deeply Learned Autoencoders. Pac Symp Biocomput. 2017;22:207-218. doi: 10.1142/9789813207813_0021. PMID: 27896976; PMCID: PMC5144587.

Thakore NJ, Lapin BR, Pioro EP; Pooled Resource Open-Access ALS Clinical Trials Consortium. Trajectories of impairment in amyotrophic lateral sclerosis: Insights from the Pooled Resource Open-Access ALS Clinical Trials cohort. Muscle Nerve. 2018 Jun;57(6):937-945. doi: 10.1002/mus.26042. Epub 2018 Jan 11. PMID: 29244213.

Shang J, Jiang M, Tong W, Xiao J, Peng J, Han J; Pooled Resource Open-Access ALS Clinical Trials Consortium*. DPPred: An Effective Prediction Framework with Concise Discriminative Patterns. IEEE Trans Knowl Data Eng. 2018 Jul;30(7):1226-1239. doi: 10.1109/TKDE.2017.2757476. Epub 2017 Sep 28. PMID: 30745791; PMCID: PMC6368191.

Jahandideh S, Taylor AA, Beaulieu D, Keymer M, Meng L, Bian A, Atassi N, Andrews J, Ennist DL. Longitudinal modeling to predict vital capacity in amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):294-302. doi: 10.1080/21678421.2017.1418003. Epub 2017 Dec 20. PMID: 29260584.

2018

Daghlas I, Lever TE, Leary E. A retrospective investigation of the relationship between baseline covariates and rate of ALSFRS-R decline in ALS clinical trials. Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):206-211. doi: 10.1080/21678421.2017.1418001. Epub 2017 Dec 20. PMID: 29260911.

Andrews JA, Meng L, Kulke SF, Rudnicki SA, Wolff AA, Bozik ME, Malik FI, Shefner JM. Association Between Decline in Slow Vital Capacity and Respiratory Insufficiency, Use of Assisted Ventilation, Tracheostomy, or Death in Patients With Amyotrophic Lateral Sclerosis. JAMA Neurol. 2018 Jan 1;75(1):58-64. doi: 10.1001/jamaneurol.2017.3339. PMID: 29181534; PMCID: PMC5833488.

van Eijk RPA, Eijkemans MJC, Ferguson TA, Nikolakopoulos S, Veldink JH, van den Berg LH. Monitoring disease progression with plasma creatinine in amyotrophic lateral sclerosis clinical trials. J Neurol Neurosurg Psychiatry. 2018 Feb;89(2):156-161. doi: 10.1136/jnnp-2017-317077. Epub 2017 Oct 30. PMID: 29084868; PMCID: PMC5800333.

Goutman SA, Brown MB, Glass JD, Boulis NM, Johe K, Hazel T, Cudkowicz M, Atassi N, Borges L, Patil PG, Sakowski SA, Feldman EL. Long-term Phase 1/2 intraspinal stem cell transplantation outcomes in ALS. Ann Clin Transl Neurol. 2018 May 2;5(6):730-740. doi: 10.1002/acn3.567. PMID: 29928656; PMCID: PMC5989736.

Harrison D, Mehta P, van Es MA, Stommel E, Drory VE, Nefussy B, van den Berg LH, Crayle J, Bedlack R; Pooled Resource Open-Access ALS Clinical Trials Consortium. "ALS reversals": demographics, disease characteristics, treatments, and co-morbidities. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):495-499. doi: 10.1080/21678421.2018.1457059. Epub 2018 Apr 2. PMID: 29607695.

van Eijk RP, Eijkemans MJ, Rizopoulos D, van den Berg LH, Nikolakopoulos S. Comparing methods to combine functional loss and mortality in clinical trials for amyotrophic lateral sclerosis. Clin Epidemiol. 2018 Mar 19;10:333-341. doi: 10.2147/CLEP.S153196. PMID: 29593436; PMCID: PMC5865572.

Karanevich AG, Statland JM, Gajewski BJ, He J. Using an onset-anchored Bayesian hierarchical model to improve predictions for amyotrophic lateral sclerosis disease progression. BMC Med Res Methodol. 2018 Feb 6;18(1):19. doi: 10.1186/s12874-018-0479-9. PMID: 29409450; PMCID: PMC5801819.

Paganoni S, Nicholson K, Chan J, Shui A, Schoenfeld D, Sherman A, Berry J, Cudkowicz M, Atassi N; Pooled Resource Open-Access ALS Clinical Trials Consortium. Urate levels predict survival in amyotrophic lateral sclerosis: Analysis of the expanded Pooled Resource Open-Access ALS clinical trials database. Muscle Nerve. 2018 Mar;57(3):430-434. doi: 10.1002/mus.25950. Epub 2017 Sep 21. PMID: 28857199; PMCID: PMC5812805.

Berry JD, Taylor AA, Beaulieu D, Meng L, Bian A, Andrews J, Keymer M, Ennist DL, Ravina B. Improved stratification of ALS clinical trials using predicted survival. Ann Clin Transl Neurol. 2018 Mar 9;5(4):474-485. doi: 10.1002/acn3.550. PMID: 29687024; PMCID: PMC5899911.

Thakore NJ, Lapin BR, Kinzy TG, Pioro EP. Deconstructing progression of amyotrophic lateral sclerosis in stages: a Markov modeling approach. Amyotroph Lateral Scler Frontotemporal Degener. 2018 Nov;19(7-8):483-494. doi: 10.1080/21678421.2018.1484925. Epub 2018 Jul 12. PMID: 30001159.

2019

Ahangaran M, Jahed-Motlagh MR, Minaei-Bidgoli B. Causal discovery from sequential data in ALS disease based on entropy criteria. J Biomed Inform. 2019 Jan;89:41-55. doi: 10.1016/j.jbi.2018.10.004. Epub 2018 Oct 16. PMID: 30339928.

Tang M, Gao C, Goutman SA, Kalinin A, Mukherjee B, Guan Y, Dinov ID. Model-Based and Model-Free Techniques for Amyotrophic Lateral Sclerosis Diagnostic Prediction and Patient Clustering. Neuroinformatics. 2019 Jul;17(3):407-421. doi: 10.1007/s12021-018-9406-9. PMID: 30460455; PMCID: PMC6527505.

Kueffner R, Zach N, Bronfeld M, Norel R, Atassi N, Balagurusamy V, Di Camillo B, Chio A, Cudkowicz M, Dillenberger D, Garcia-Garcia J, Hardiman O, Hoff B, Knight J, Leitner ML, Li G, Mangravite L, Norman T, Wang L; ALS Stratification Consortium, Xiao J, Fang WC, Peng J, Yang C, Chang HJ, Stolovitzky G. Stratification of amyotrophic lateral sclerosis patients: a crowdsourcing approach. Sci Rep. 2019 Jan 24;9(1):690. doi: 10.1038/s41598-018-36873-4. PMID: 30679616; PMCID: PMC6345935.

Ackrivo J, Hansen-Flaschen J, Wileyto EP, Schwab RJ, Elman L, Kawut SM. Development of a prognostic model of respiratory insufficiency or death in amyotrophic lateral sclerosis. Eur Respir J. 2019 Apr 18;53(4):1802237. doi: 10.1183/13993003.02237-2018. PMID: 30728207; PMCID: PMC6684229.

Zandonà A, Vasta R, Chiò A, Di Camillo B. A Dynamic Bayesian Network model for the simulation of Amyotrophic Lateral Sclerosis progression. BMC Bioinformatics. 2019 Apr 18;20(Suppl 4):118. doi: 10.1186/s12859-019-2692-x. PMID: 30999865; PMCID: PMC6471677.

Grollemund V, Pradat PF, Querin G, Delbot F, Le Chat G, Pradat-Peyre JF, Bede P. Machine Learning in Amyotrophic Lateral Sclerosis: Achievements, Pitfalls, and Future Directions. Front Neurosci. 2019 Feb 28;13:135. doi: 10.3389/fnins.2019.00135. PMID: 30872992; PMCID: PMC6403867.

Thakore NJ, Lapin BR, Pioro EP, Aboussouan LS. Variation in noninvasive ventilation use in amyotrophic lateral sclerosis. Neurology. 2019 Jul 16;93(3):e306-e316. doi: 10.1212/WNL.0000000000007776. Epub 2019 Jun 10. PMID: 31182506.

Schoenfeld DA, Finkelstein DM, Macklin E, Zach N, Ennist DL, Taylor AA, Atassi N; Pooled Resource Open-Access ALS Clinical Trials Consortium. Design and analysis of a clinical trial using previous trials as historical control. Clin Trials. 2019 Oct;16(5):531-538. doi: 10.1177/1740774519858914. Epub 2019 Jul 1. PMID: 31256630; PMCID: PMC7008967.

van Eijk RPA, Bakers JNE, van Es MA, Eijkemans MJC, van den Berg LH. Implications of spirometric reference values for amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2019 Nov;20(7-8):473-480. doi: 10.1080/21678421.2019.1634736. Epub 2019 Jul 4. PMID: 31271047.

Gordon J, Lerner B. Insights into Amyotrophic Lateral Sclerosis from a Machine Learning Perspective. J Clin Med. 2019 Oct 1;8(10):1578. doi: 10.3390/jcm8101578. PMID: 31581566; PMCID: PMC6832919.

Huber C, Benda N, Friede T. A comparison of subgroup identification methods in clinical drug development: Simulation study and regulatory considerations. Pharm Stat. 2019 Oct;18(5):600-626. doi: 10.1002/pst.1951. Epub 2019 Jul 3. PMID: 31270933; PMCID: PMC6772173.

van Eijk RPA, Nikolakopoulos S, Roes KCB, Middelkoop BM, Ferguson TA, Shaw PJ, Leigh PN, Al-Chalabi A, Eijkemans MJC, van den Berg LH. Critical design considerations for time-to-event endpoints in amyotrophic lateral sclerosis clinical trials. J Neurol Neurosurg Psychiatry. 2019 Dec;90(12):1331-1337. doi: 10.1136/jnnp-2019-320998. Epub 2019 Jul 10. PMID: 31292200; PMCID: PMC6902062.

Patterns of VC Decline in Amyotrophic Lateral Sclerosis. A More Robust Prognostication? Ahmet Baydur Am J Respir Crit Care Med. 2019 Dec 15; 200(12): 1461–1462. Published online 2019 Dec 15. doi: 10.1164/rccm.201907-1440ED PMCID: PMC6909827

Ackrivo J, Hansen-Flaschen J, Jones BL, Wileyto EP, Schwab RJ, Elman L, Kawut SM. Classifying Patients with Amyotrophic Lateral Sclerosis by Changes in FVC. A Group-based Trajectory Analysis. Am J Respir Crit Care Med. 2019 Dec 15;200(12):1513-1521. doi: 10.1164/rccm.201902-0344OC. PMID: 31322417; PMCID: PMC6909832.

2020

Thakore NJ, Lapin BR, Pioro EP; Pooled Resource Open-Access Als Clinical Trials Consortium. Stage-specific riluzole effect in amyotrophic lateral sclerosis: a retrospective study. Amyotroph Lateral Scler Frontotemporal Degener. 2020 Feb;21(1-2):140-143. doi: 10.1080/21678421.2019.1655060. Epub 2019 Aug 27. PMID: 31450993.

Braun N, Macklin EA, Sinani E, Sherman A, Weber M; Pooled Resource Open-Access ALS Clinical Trials Consortium. The revised El Escorial criteria "clinically probable laboratory supported ALS"-once a promising now a superfluous category? Amyotroph Lateral Scler Frontotemporal Degener. 2020 Feb;21(1-2):24-28. doi: 10.1080/21678421.2019.1666875. Epub 2019 Sep 27. PMID: 31561715.

Zhou N, Manser P. Does including machine learning predictions in ALS clinical trial analysis improve statistical power? Ann Clin Transl Neurol. 2020 Oct;7(10):1756-1765. doi: 10.1002/acn3.51140. Epub 2020 Aug 30. PMID: 32862509; PMCID: PMC7545604.

Prell T, Grosskreutz J; Pooled Resource Open-Access ALS Clinical Trials Consortium. Use of vitamins by participants in amyotrophic lateral sclerosis clinical trials. PLoS One. 2020 Aug 13;15(8):e0237175. doi: 10.1371/journal.pone.0237175. PMID: 32790757; PMCID: PMC7425848.

Fournier CN, Bedlack R, Quinn C, Russell J, Beckwith D, Kaminski KH, Tyor W, Hertzberg V, James V, Polak M, Glass JD. Development and Validation of the Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS). JAMA Neurol. 2020 Apr 1;77(4):480-488. doi: 10.1001/jamaneurol.2019.4490. PMID: 31886839; PMCID: PMC6990811.

Korepanova N, Seibold H, Steffen V, Hothorn T. Survival forests under test: Impact of the proportional hazards assumption on prognostic and predictive forests for amyotrophic lateral sclerosis survival. Stat Methods Med Res. 2020 May;29(5):1403-1419. doi: 10.1177/0962280219862586. Epub 2019 Jul 15. PMID: 31304888.

Ahangaran M, Jahed-Motlagh MR, Minaei-Bidgoli B. A novel method for predicting the progression rate of ALS disease based on automatic generation of probabilistic causal chains. Artif Intell Med. 2020 Jul;107:101879. doi: 10.1016/j.artmed.2020.101879. Epub 2020 May 22. PMID: 32828438.

van Eijk RPA. Frequent self-assessments in ALS Clinical Trials: worthwhile or an unnecessary burden for patients? Ann Clin Transl Neurol. 2020 Oct;7(10):2074-2075. doi: 10.1002/acn3.51178. Epub 2020 Sep 18. PMID: 32946644; PMCID: PMC7545614.

Grollemund V, Chat GL, Secchi-Buhour MS, Delbot F, Pradat-Peyre JF, Bede P, Pradat PF. Development and validation of a 1-year survival prognosis estimation model for Amyotrophic Lateral Sclerosis using manifold learning algorithm UMAP. Sci Rep. 2020 Aug 7;10(1):13378. doi: 10.1038/s41598-020-70125-8. PMID: 32770027; PMCID: PMC7414917.

Barczewska M, Maksymowicz S, Zdolińska-Malinowska I, Siwek T, Grudniak M. Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study. Stem Cell Rev Rep. 2020 Oct;16(5):922-932. doi: 10.1007/s12015-020-10016-7. PMID: 32725316; PMCID: PMC7456414.

de Jongh AD, van den Berg LH, van Eijk RPA. Reconsidering the revised amyotrophic lateral sclerosis functional rating scale for ALS clinical trials. J Neurol Neurosurg Psychiatry. 2021 May;92(5):569-570. doi: 10.1136/jnnp-2020-325253. Epub 2020 Nov 23. PMID: 33229452.

Tavazzi, E., Daberdaku, S., Vasta, R. et al. Exploiting mutual information for the imputation of static and dynamic mixed-type clinical data with an adaptive k-nearest neighbours approach. BMC Med Inform Decis Mak 20, 174 (2020). https://doi.org/10.1186/s12911-020-01166-2

Saitoh Y, Takahashi Y. Riluzole for the treatment of amyotrophic lateral sclerosis. Neurodegener Dis Manag. 2020 Dec;10(6):343-355. doi: 10.2217/nmt-2020-0033. Epub 2020 Aug 27. PMID: 32847483.

Farhad Hatami, Konstantinos Perrakis, Johnathan Cooper-Knock, Sach Mukherjee, Frank Dondelinger. Penalized longitudinal mixed models with latent group structure, with an application in neurodegenerative diseases. medRxiv 2020.11.10.20229302; doi: https://doi.org/10.1101/2020.11.10.20229302

2021

Kiernan MC, Vucic S, Talbot K, McDermott CJ, Hardiman O, Shefner JM, Al-Chalabi A, Huynh W, Cudkowicz M, Talman P, Van den Berg LH, Dharmadasa T, Wicks P, Reilly C, Turner MR. Improving clinical trial outcomes in amyotrophic lateral sclerosis. Nat Rev Neurol. 2021 Feb;17(2):104-118. doi: 10.1038/s41582-020-00434-z. Epub 2020 Dec 18. PMID: 33340024; PMCID: PMC7747476.

Perry BJ, Nelson J, Wong JB, Kent DM. The cumulative incidence of dysphagia and dysphagia-free survival in persons diagnosed with amyotrophic lateral sclerosis. Muscle Nerve. 2021 Apr 13. doi: 10.1002/mus.27244. Epub ahead of print. PMID: 33851421.

Johannesen S, Huie JR, Budeus B, Peters S, Wirth AM, Iberl S, Kammermaier T, Kobor I, Wirkert E, Küspert S, Tahedl M, Grassinger J, Pukrop T, Schneider A, Aigner L, Schulte-Mattler W, Schuierer G, Koch W, Bruun TH, Ferguson AR, Bogdahn U. Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis. Front Neurol. 2021 Mar 18;12:616289. doi: 10.3389/fneur.2021.616289. PMID: 33815246; PMCID: PMC8012841.

ALSUNTANGLED GROUP. ALSUntangled #61: melatonin. Amyotroph Lateral Scler Frontotemporal Degener. 2021 May 18:1-4. doi: 10.1080/21678421.2021.1927103. Epub ahead of print. PMID: 34002641.

Bald EM, Nance CS, Schultz JL. Melatonin may slow disease progression in amyotrophic lateral sclerosis: Findings from the Pooled Resource Open-Access ALS Clinic Trials database. Muscle Nerve. 2021 Apr;63(4):572-576. doi: 10.1002/mus.27168. Epub 2021 Jan 24. PMID: 33428242.

Daghlas SA, Govindarajan R; Pooled Resource Open-Access ALS Clinical Trials Consortium. Relative effects of forced vital capacity and ALSFRS-R on survival in ALS. Muscle Nerve. 2021 Jun 2. doi: 10.1002/mus.27344. Epub ahead of print. PMID: 34076262.

Haji S, Sako W, Murakami N, Osaki Y, Furukawa T, Izumi Y, Kaji R. The value of serum uric acid as a prognostic biomarker in amyotrophic lateral sclerosis: Evidence from a meta-analysis. Clin Neurol Neurosurg. 2021 Apr;203:106566. doi: 10.1016/j.clineuro.2021.106566. Epub 2021 Feb 19. PMID: 33706058.

Tran TD, Lesaffre E, Verbeke G, Molenberghs G. Serial correlation structures in latent linear mixed models for analysis of multivariate longitudinal ordinal responses. Stat Med. 2021 Feb 10;40(3):578-592. doi: 10.1002/sim.8790. Epub 2020 Oct 28. PMID: 33118185.

Morgadinho J, Pronto-Laborinho AC, Conceição VA, Gromicho M, Pinto S, Swash M, de Carvalho M. Plasma Creatinine Level Does Not Predict Respiratory Function in Amyotrophic Lateral Sclerosis. J Neuromuscul Dis. 2021 Feb 23. doi: 10.3233/JND-200583. Epub ahead of print. PMID: 33646173.

Perry BJ, Nelson J, Wong JB, Kent DM; Pooled Resource Open-Access ALS Clinical Trials Consortium. Predicting dysphagia onset in patients with ALS: the ALS dysphagia risk score. Amyotroph Lateral Scler Frontotemporal Degener. 2021 Aug 10:1-8. doi: 10.1080/21678421.2021.1961805. Epub ahead of print. PMID: 34375156.

van Eijk RPA, Nikolakopoulos S, Roes KCB, Kendall L, Han SS, Lavrov A, Epstein N, Kliest T, de Jongh AD, Westeneng HJ, Al-Chalabi A, Van Damme P, Hardiman O, Shaw PJ, McDermott CJ, Eijkemans MJC, van den Berg LH. Innovating Clinical Trials for Amyotrophic Lateral Sclerosis: Challenging the Established Order. Neurology. 2021 Sep 14;97(11):528-536. doi: 10.1212/WNL.0000000000012545. Epub 2021 Jul 27. PMID: 34315786; PMCID: PMC8456357.

Introna A, Milella G, Morea A, Ucci M, Fraddosio A, Zoccolella S, D'Errico E, Simone IL. King's college progression rate at first clinical evaluation: A new measure of disease progression in amyotrophic lateral sclerosis. J Neurol Sci. 2021 Dec 15;431:120041. doi: 10.1016/j.jns.2021.120041. Epub 2021 Oct 28. PMID: 34736124.

Dalgıç ÖO, Wu H, Safa Erenay F, Sir MY, Özaltın OY, Crum BA, Pasupathy KS; Pooled Resource Open-Access ALS Clinical Trials Consortium. Mapping of critical events in disease progression through binary classification: Application to amyotrophic lateral sclerosis. J Biomed Inform. 2021 Nov;123:103895. doi: 10.1016/j.jbi.2021.103895. Epub 2021 Aug 25. PMID: 34450286.

2022

Leone MA, Mandrioli J, Russo S, Cucovici A, Gianferrari G, Lisnic V, Muresanu DF, Giuliani F, Copetti M, The Pooled Resource Open-Access Als Clinical Trials Consortium, Fontana A. Neutrophils-to-Lymphocyte Ratio Is Associated with Progression and Overall Survival in Amyotrophic Lateral Sclerosis. Biomedicines. 2022 Feb 1;10(2):354. doi: 10.3390/biomedicines10020354. PMID: 35203564; PMCID: PMC8962424.

Balendra R, Jones AR, Al Khleifat A, Chiwera T, Wicks P, Young CA, Shaw PJ, Turner MR, Leigh PN, Al-Chalabi A. Comparison Of King's Clinical Staging In Multinational Amyotrophic Lateral Sclerosis Cohorts. Amyotroph Lateral Scler Frontotemporal Degener. 2023 Feb;24(1-2):71-81. doi: 10.1080/21678421.2022.2090847. Epub 2022 Jul 19. PMID: 35852375.

Pancotti C, Birolo G, Rollo C, Sanavia T, Di Camillo B, Manera U, Chiò A, Fariselli P. Deep learning methods to predict amyotrophic lateral sclerosis disease progression. Sci Rep. 2022 Aug 12;12(1):13738. doi: 10.1038/s41598-022-17805-9. PMID: 35962027; PMCID: PMC9374680.

Kobayakawa Y, Todaka K, Hashimoto Y, Ko S, Shiraishi W, Kishimoto J, Kira JI, Yamasaki R, Isobe N; Pooled Resource Open-Access ALS Clinical Trials Consortium. A novel quantitative indicator for disease progression rate in amyotrophic lateral sclerosis. J Neurol Sci. 2022 Nov 15;442:120389. doi: 10.1016/j.jns.2022.120389. Epub 2022 Aug 24. PMID: 36041329.

Thakore NJ, Lapin BR, Mitsumoto H, Pooled Resource Open-Access Als Clinical Trials Consortium. Early initiation of riluzole may improve absolute survival in amyotrophic lateral sclerosis. Muscle Nerve. 2022 Dec;66(6):702-708. doi: 10.1002/mus.27724. Epub 2022 Oct 4. PMID: 36117390; PMCID: PMC9828202.

Song Y, Cheng H, Liu J, Kazuo S, Feng L, Wei Y, Zhang C, Gao Y; Pooled Resource Open-Access ALS Clinical Trials Consortium. Effectiveness of herbal medicine on patients with amyotrophic lateral sclerosis: Analysis of the PRO-ACT data using propensity score matching. Phytomedicine. 2022 Dec;107:154461. doi: 10.1016/j.phymed.2022.154461. Epub 2022 Sep 18. PMID: 36198223.

2023

Tavazzi E, Gatta R, Vallati M, Cotti Piccinelli S, Filosto M, Padovani A, Castellano M, Di Camillo B. Leveraging process mining for modeling progression trajectories in amyotrophic lateral sclerosis. BMC Med Inform Decis Mak. 2023 Feb 2;22(Suppl 6):346. doi: 10.1186/s12911-023-02113-7. PMID: 36732801; PMCID: PMC9896660.

Zoccolella S, Milella G, Giugno A, Urso D, Tamburrino L, Nigro S, Gnoni V, Filardi M, Logroscino G. Split-elbow sign in the PRO-ACT and Southern Italy ALS cohorts: a potential marker of disease severity and lower motor neuron involvement? J Neurol. 2023 Mar 14. doi: 10.1007/s00415-023-11660-y. Epub ahead of print. PMID: 36917342.

Huang B, Geng X, Yu Z, Zhang C, Chen Z. Dynamic effects of prognostic factors and individual survival prediction for amyotrophic lateral sclerosis disease. Ann Clin Transl Neurol. 2023 Apr 4. doi: 10.1002/acn3.51771. Epub ahead of print. PMID: 37014017.

Morimoto S, Takahashi S, Ito D, Daté Y, Okada K, Kato C, Nakamura S, Ozawa F, Chyi CM, Nishiyama A, Suzuki N, Fujimori K, Kondo T, Takao M, Hirai M, Kabe Y, Suematsu M, Jinzaki M, Aoki M, Fujiki Y, Sato Y, Suzuki N, Nakahara J; Pooled Resource Open-Access ALS Clinical Trials Consortium; Okano H. Phase 1/2a clinical trial in ALS with ropinirole, a drug candidate identified by iPSC drug discovery. Cell Stem Cell. 2023 Jun 1;30(6):766-780.e9. doi: 10.1016/j.stem.2023.04.017. PMID: 37267913.

Nam JY, Chun S, Lee TY, Seo Y, Kim K, Park J, Sung W, Oh KW, Lee S, Park JS, Oh J, Chung KC, An H, Chu HS, Son B, Kim SH. Long-term survival benefits of intrathecal autologous bone marrow-derived mesenchymal stem cells (Neuronata-R®: lenzumestrocel) treatment in ALS: Propensity-score-matched control, surveillance study. Front Aging Neurosci. 2023 Apr 12;15:1148444. doi: 10.3389/fnagi.2023.1148444. PMID: 37122380; PMCID: PMC10130504.

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