Efficacy and tolerability of EH301 for amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled human pilot study
Open accessAbstract
BACKGROUND: Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease, characterized by progressive loss of spinal and cortical motor neurons, leading to muscular atrophy, respiratory failure, and ultimately death. There is no known cure, and the clinical benefit of the two drugs approved to treat ALS remains unclear. Novel disease-modifying therapeutics that are able to modulate the disease course are desperately needed. Our objective was to evaluate the efficacy and tolerability of Elysium Health's candidate drug EH301 in people with ALS (PALS). METHODS: This was a single-center, prospective, double-blind, randomized, placebo-controlled pilot study. Thirty-two PALS were recruited thanks to the collaboration of the Spanish Foundation for ALS Research (FUNDELA). Study participants were randomized to receive either EH301 or placebo and underwent evaluation for 4 months. Differences between EH301 and placebo-treated participants were evaluated based on standard clinical endpoints, including the revised ALS functional rating scale (ALSFRS-R), forced vital capacity (FVC), and the Medical Research Council (MRC) grading scale. RESULTS: Compared to placebo, participants treated with EH301 demonstrated significant improvements in the ALSFRS-R score, pulmonary function, muscular strength, and in skeletal muscle/fat weight ratio. EH301 was shown to significantly slow the progression of ALS relative to placebo, and even showed improvements in several key outcome measures compared with baseline. CONCLUSIONS: This study provides evidence in support of the disease-modifying effects of EH301 for the treatment of ALS.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
A preliminary EH301 trial, using an NAD+ precursor plus a SIRT1 activator, supports the importance of NAD+ metabolism and SIRT1 activation.
“The importance of NAD+ metabolism and SIRT1 activation has been supported by a recent preliminary clinical trial with EH301, consisting of an NAD+ precursor and a SIRT1 activator”
From NAD+ in Brain Aging and Neurodegenerative Disorders (2019) · cites this paper as de la Rubia et al., 2019
Despite the small number of participants, the EH301 trial supports the potential of NAD+ augmentation or SIRT1 activation as an ALS treatment and warrants larger trials.
“Despite the low level of participants in this study, it sets the stage for the potential for NAD+ augmentation/SIRT1 activation as a treatment for ALS, and large-scale phase trials are warranted.”
From NAD+ in Brain Aging and Neurodegenerative Disorders (2019) · cites this paper as de la Rubia et al., 2019
The ALS EH301 trial was randomized and double-blind and included both males and females.
“The study was a randomized double-blind design and included both males and females.”
From NAD+ in Brain Aging and Neurodegenerative Disorders (2019) · cites this paper as de la Rubia et al., 2019
NRPT/EH301 treatment improved ALSFRS-R, increased the MRC grading scale index, forced vital capacity, and muscle electrical activity in ALS patients.
“NRPT/EH301 treatment improved ALS functional rating scale revised (ALSFRS-R), increased the MRC grading scale index (grades muscle power), and forced vital capacity (FVC, functional respiratory capacity test), as well as increased electrical activity in muscles.”
From NAD+ in Brain Aging and Neurodegenerative Disorders (2019) · cites this paper as de la Rubia et al., 2019
After 4 months, all patients in the EH301 group chose to continue treatment; after 1 year, no deterioration except in FVC was observed.
“All patients in the EH301 group choose to continue the treatment after 4 months, and thus the treatment was finalized after 1-year treatment, where no deterioration, except of FVC, was observed”
From NAD+ in Brain Aging and Neurodegenerative Disorders (2019) · cites this paper as de la Rubia et al., 2019
One trial showed short-term NR administration has some beneficial effects in healthy elderly individuals, and another showed positive results in patients with ALS.
“One trial showed that short-term NR administration has some beneficial effects in healthy elderly individuals185, and another showed positive results in patients with ALS93,186,187.”
From NAD+ metabolism and its roles in cellular processes during ageing (2021) · cites this paper as [187]
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
El Escorial revisited: revised criteria for the diagnosis of amyotrophic lateral sclerosis
Brooks, B. R., Miller, R. G., Swash, M., & Munsat, T. L. (2000) · Amyotrophic Lateral Sclerosis and Other Motor Neuron Disorders · doi:10.1080/146608200300079536 · Cited by 5,433 · see citing works
Nutrient control of glucose homeostasis through a complex of PGC-1α and SIRT1
Rodgers, J. T., Lerin, C., Haas, W., Gygi, S. P., Spiegelman, B. M., & Puigserver, P. (2005) · Nature · doi:10.1038/nature03354 · Cited by 3,160 · see citing works
Calorie restriction reduces oxidative stress by SIRT3-mediated SOD2 activation
Qiu, X., Brown, K., Hirschey, M. D., Verdin, E., & Chen, D. (2010) · Cell Metabolism · doi:10.1016/j.cmet.2010.11.015 · Cited by 1,310 · see citing works
Definition of the Mediterranean Diet; A Literature Review
Davis, C. R., Bryan, J., Hodgson, J. M., & Murphy, K. J. (2015) · Nutrients · doi:10.3390/nu7115459 · Cited by 1,200 · see citing works
Abe, K., Aoki, M., Tsuji, S., Itoyama, Y., Sobue, G., Togo, M., Hamada, C., Tanaka, M., Akimoto, M., Nakamura, K., et al. (2017) · The Lancet Neurology · doi:10.1016/s1474-4422(17)30115-1 · Cited by 942 · see citing works
SARM1 activation triggers axon degeneration locally via NAD <sup>+</sup> destruction
Gerdts, J., Brace, E. J., Sasaki, Y., DiAntonio, A., & Milbrandt, J. (2015) · Science · doi:10.1126/science.1258366 · Cited by 593 · see citing works
Fang, E. F., Kassahun, H., Croteau, D. L., Scheibye-Knudsen, M., Marosi, K., Lu, H., Shamanna, R. A., Kalyanasundaram, S., Bollineni, R. C., Wilson, M. A., et al. (2016) · Cell Metabolism · doi:10.1016/j.cmet.2016.09.004 · Cited by 575 · see citing works
Calorie restriction and sirtuins revisited
Guarente, L. (2013) · Genes & Development · doi:10.1101/gad.227439.113 · Cited by 464 · see citing works
Self-citationThe Role of PARP-1 and PARP-2 Enzymes in Metabolic Regulation and Disease
Bai, P., & Cantó, C. (2012) · Cell Metabolism · doi:10.1016/j.cmet.2012.06.016 · Cited by 312 · see citing works
Charcot, J. M. (1869) · Medical Entomology and Zoology · Cited by 305 · see citing works
Czapliński, A. (2005) · Journal of Neurology Neurosurgery & Psychiatry · doi:10.1136/jnnp.2005.072660 · Cited by 282 · see citing works
Anthropometry in Body Composition: An Overview
Wang, J., Thornton, J. C., KOLESNIK, S., & Pierson, R. N. (2000) · Annals of the New York Academy of Sciences · doi:10.1111/j.1749-6632.2000.tb06474.x · Cited by 276 · see citing works
Nrf2/ARE-mediated antioxidant actions of pro-electrophilic drugs
Satoh, T., McKercher, S. R., & Lipton, S. A. (2013) · Free Radical Biology and Medicine · doi:10.1016/j.freeradbiomed.2013.07.022 · Cited by 267 · see citing works
ALSFRS-R score and its ratio: A useful predictor for ALS-progression
Kollewe, K., Mauss, U., Krampfl, K., Petri, S., Dengler, R., & Mohammadi, B. (2008) · Journal of the Neurological Sciences · doi:10.1016/j.jns.2008.07.016 · Cited by 253 · see citing works
Cudkowicz, M., Titus, S., Kearney, M., Yu, H., Sherman, A., Schoenfeld, D., Hayden, D., Shui, A. M., Brooks, B. R., CONWIT, R. A., et al. (2014) · The Lancet Neurology · doi:10.1016/s1474-4422(14)70222-4 · Cited by 243 · see citing works
Dellinger, R. W., Santos, S. R., Morris, M., Evans, M., Alminana, D., Guarente, L., & Marcotulli, E. (2017) · npj Aging and Mechanisms of Disease · doi:10.1038/s41514-017-0016-9 · Cited by 181 · see citing works
Self-citationClinical significance in the change of decline in ALSFRS-R
Castrillo‐Viguera, C., Grasso, D. L., Simpson, E., Shefner, J. M., & Cudkowicz, M. (2010) · Amyotrophic Lateral Sclerosis · doi:10.3109/17482960903093710 · Cited by 121 · see citing works
Cheng, Y., Di, S., Fan, C., Cai, L., Gao, C., Jiang, P., Hu, W., Ma, Z., Jiang, S., Dong, Y., et al. (2016) · APOPTOSIS · doi:10.1007/s10495-016-1258-x · Cited by 91 · see citing works
Resveratrol upregulated heat shock proteins and extended the survival of G93A-SOD1 mice
Han, S.-Y., Choi, J.-R., Shin, K. S., & Kang, S. J. (2012) · Brain Research · doi:10.1016/j.brainres.2012.09.022 · Cited by 83 · see citing works
Spirometry. Spanish Society of Pulmonology and Thoracic Surgery (SEPAR).
García-Río, F., Calle, M., Burgos, F., Casan, P., del Campo, F., Galdiz, J., Giner, J., González-Mangado, N., Ortega, F., & Maestú, L. P. (2013) · Archivos de Bronconeumologia · doi:10.1016/j.arbres.2013.04.001 · Cited by 75 · see citing works
Pharmacokinetics and tissue distribution of pterostilbene in the rat
Azzolini, M., Spina, M. L., Mattarei, A., Paradisi, C., Zoratti, M., & Biasutto, L. (2014) · Molecular Nutrition & Food Research · doi:10.1002/mnfr.201400244 · Cited by 69 · see citing works
Benlloch, M., Obrador, E., Vallés, S. L., Rodríguez, M. L., Sirerol, J. A., Alcácer, J., Pellicer, J. A., Palmer, R. S., Cerdá, C., Sáez, G. T., et al. (2015) · Antioxidants and Redox Signaling · doi:10.1089/ars.2015.6437 · Cited by 66 · see citing works
Self-citationPinto, S., & de Carvalho, M. A. (2016) · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · doi:10.1080/21678421.2016.1249486 · Cited by 66 · see citing works
Wilkins, H., Kirchhof, D., Manning, E., Joseph, J. W., & Linseman, D. A. (2013) · Journal of Biological Chemistry · doi:10.1074/jbc.m112.405738 · Cited by 65 · see citing works
Körner, S., Böselt, S., Thau, N., Rath, K. J., Dengler, R., & Petri, S. (2013) · Neurodegenerative Diseases · doi:10.1159/000338048 · Cited by 64 · see citing works
Guo, Y., Zhang, L., Li, F., Hu, C., & Zhang, Z. (2016) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2016.02.052 · Cited by 62 · see citing works
Quantitating progression in ALS
de Carvalho, M. A., Scotto, M. G., Lopes, A., & Swash, M. (2005) · Neurology · doi:10.1212/01.wnl.0000162036.76024.ab · Cited by 61 · see citing works
Wang, B., Liu, H., Yue, L., Li, X., Zhao, L., Yang, X., Wang, X., Yang, Y., & Qu, Y. (2016) · Brain Research · doi:10.1016/j.brainres.2016.04.048 · Cited by 59 · see citing works
Could sirtuin activities modify ALS onset and progression?
Tang, B. L. (2017) · Cellular and Molecular Neurobiology · doi:10.1007/s10571-016-0452-2 · Cited by 24 · see citing works
Discussing edaravone with the ALS patient: an ethical framework from a U.S. perspective
Yeo, C. J. J., & Simmons, Z. (2018) · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · doi:10.1080/21678421.2018.1425455 · Cited by 17 · see citing works
Flagged first, then by citations
Cited by
Sorted by citations
NAD+ metabolism and its roles in cellular processes during ageing
Covarrubias, A. J., Perrone, R., Grozio, A., & Verdin, E. (2021) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-020-00313-x · Cited by 1,617 · see citing works
NAD+ metabolism: pathophysiologic mechanisms and therapeutic potential
Xie, N., Zhang, L., Gao, W. Q., Huang, C., Huber, P., Zhou, X., Li, C. L., Shen, G., & Zou, B. (2020) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-020-00311-7 · Cited by 1,078 · see citing works
NAD+ in Brain Aging and Neurodegenerative Disorders
Lautrup, S., Sinclair, D. A., Mattson, M. P., & Fang, E. F. (2019) · Cell Metabolism · doi:10.1016/j.cmet.2019.09.001 · Cited by 790 · see citing works
NAD+ homeostasis in health and disease
Katsyuba, E., Romani, M., Hofer, D., & Auwerx, J. (2020) · Nature Metabolism · doi:10.1038/s42255-019-0161-5 · Cited by 627 · see citing works
Human trials exploring anti-aging medicines
Guarente, L., Sinclair, D. A., & Kroemer, G. (2024) · Cell Metabolism · doi:10.1016/j.cmet.2023.12.007 · Cited by 257 · see citing works
Self-citationCunha‐Oliveira, T., Montezinho, L., Mendes, C., Firuzi, O., Saso, L., Oliveira, P. J., & Silva, F. S. G. (2020) · Oxidative Medicine and Cellular Longevity · doi:10.1155/2020/5021694 · Cited by 214 · see citing works
McReynolds, M. R., Chellappa, K., & Baur, J. A. (2020) · Experimental Gerontology · doi:10.1016/j.exger.2020.110888 · Cited by 169 · see citing works
Regulation of and challenges in targeting NAD+ metabolism
Migaud, M. E., Ziegler, M., & Baur, J. A. (2024) · Nature Reviews Molecular Cell Biology · doi:10.1038/s41580-024-00752-w · Cited by 139 · see citing works
Obrador, E., Palmer, R. S., López‐Blanch, R., Jihad-Jebbar, A., Vallés, S. L., & Estrela, J. M. (2020) · Antioxidants · doi:10.3390/antiox9090901 · Cited by 129 · see citing works
Self-citationHow gut microbes could drive brain disorders
Willyard, C. (2021) · Nature · doi:10.1038/d41586-021-00260-3 · Cited by 124 · see citing works
CNS glucose metabolism in Amyotrophic Lateral Sclerosis: a therapeutic target?
Tefera, T. W., Steyn, F. J., Ngo, S. T., & Borges, K. (2021) · Cell & Bioscience · doi:10.1186/s13578-020-00511-2 · Cited by 118 · see citing works
Constitutively active SARM1 variants that induce neuropathy are enriched in ALS patients
Bloom, A. J., Mao, X. R., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Molecular Neurodegeneration · doi:10.1186/s13024-021-00511-x · Cited by 118 · see citing works
Obrador, E., Palmer, R. S., López‐Blanch, R., Jihad-Jebbar, A., Vallés, S. L., & Estrela, J. M. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22126352 · Cited by 104 · see citing works
Self-citationWhat is really known about the effects of nicotinamide riboside supplementation in humans
Damgaard, M. V., & Treebak, J. T. (2023) · Science Advances · doi:10.1126/sciadv.adi4862 · Cited by 99 · see citing works
ALS motor neurons exhibit hallmark metabolic defects that are rescued by SIRT3 activation
Hor, J., Santosa, M. M., Lim, V. J. W., Ho, B. X., Taylor, A., Khong, Z. J., Ravits, J., Fan, Y., Liou, Y., Soh, B.-S., et al. (2020) · Cell Death and Differentiation · doi:10.1038/s41418-020-00664-0 · Cited by 83 · see citing works
Zhou, Z., & Tan, E. K. (2020) · Ageing Research Reviews · doi:10.1016/j.arr.2020.101107 · Cited by 76 · see citing works
Harlan, B. A., Killoy, K. M., Pehar, M., Liu, L., Auwerx, J. H., & Vargas, M. R. (2020) · Experimental Neurology · doi:10.1016/j.expneurol.2020.113219 · Cited by 70 · see citing works
New horizons in life extension, healthspan extension and exceptional longevity
Couteur, D. G. L., & Barzilai, N. (2022) · Age and Ageing · doi:10.1093/ageing/afac156 · Cited by 63 · see citing works
The balance between NAD+ biosynthesis and consumption in ageing
Strømland, Ø., Diab, J., Ferrario, E., Sverkeli, L. J., & Ziegler, M. (2021) · Mechanisms of Ageing and Development · doi:10.1016/j.mad.2021.111569 · Cited by 63 · see citing works
Freeberg, K. A., Udovich, C. C., Martens, C. R., Seals, D. R., & Craighead, D. H. (2023) · The Journals of Gerontology Series A · doi:10.1093/gerona/glad106 · Cited by 68 · see citing works
Clinical Evidence for Targeting NAD Therapeutically
Radenković, D., Reason, & Verdin, E. M. (2020) · Pharmaceuticals · doi:10.3390/ph13090247 · Cited by 55 · see citing works
Morató, L., Astori, S., Zalachoras, I., Rodrigues, J., Ghosal, S., Huang, W., de Suduiraut, I. G., Grosse, J., Zanoletti, O., Cao, L., et al. (2022) · Science Advances · doi:10.1126/sciadv.abj9109 · Cited by 51 · see citing works
The panoramic view of amyotrophic lateral sclerosis: A fatal intricate neurological disorder
Dhasmana, S., Dhasmana, A., Narula, A. S., Jaggi, M., Yallapu, M. M., & Chauhan, S. C. (2021) · Life Sciences · doi:10.1016/j.lfs.2021.120156 · Cited by 52 · see citing works
Emerging strategies, applications and challenges of targeting NAD+ in the clinic
Zhang, J., Wang, H., Lautrup, S., Nilsen, H. L., Treebak, J. T., Watne, L. O., Selbæk, G., Wu, L. E., Omland, T., Pirinen, E., et al. (2025) · Nature Aging · doi:10.1038/s43587-025-00947-6 · Cited by 58 · see citing works
NAD+ precursors protect corneal endothelial cells from UVB-induced apoptosis
Zhao, C., Li, W., Duan, H., Li, Z., Jia, Y., Zhang, S., Wang, X., Zhou, Q., & Shi, W. (2020) · American Journal of Physiology-Cell Physiology · doi:10.1152/ajpcell.00445.2019 · Cited by 47 · see citing works
The crosstalk of NAD, ROS and autophagy in cellular health and ageing
Sedlackova, L., & Korolchuk, V. I. (2020) · Biogerontology · doi:10.1007/s10522-020-09864-0 · Cited by 47 · see citing works
Obrador, E., Palmer, R. S., Marchio, P., López‐Blanch, R., Jihad-Jebbar, A., Rivera, P., Vallés, S. L., Banacloche, S., Alcácer, J., Colomer, N., et al. (2020) · Molecular Neurobiology · doi:10.1007/s12035-020-02188-7 · Cited by 46 · see citing works
Self-citationNicotinamide Adenine Dinucleotide in Aging Biology: Potential Applications and Many Unknowns
Bhasin, S., Seals, D. R., Migaud, M. E., Musi, N., & Baur, J. A. (2023) · Endocrine Reviews · doi:10.1210/endrev/bnad019 · Cited by 47 · see citing works
Gilmour, B. C., Gudmundsrud, R., Frank, J., Hov, A., Lautrup, S., Aman, Y., Røsjø, H. R., Brenner, C., Ziegler, M., Tysnes, O., et al. (2020) · Mechanisms of Ageing and Development · doi:10.1016/j.mad.2020.111208 · Cited by 43 · see citing works
Gut microbiome and amyotrophic lateral sclerosis: a systematic review of current evidence
Sun, J., Huang, T., Debelius, J. W., & Fang, F. (2021) · Journal of Internal Medicine · doi:10.1111/joim.13336 · Cited by 43 · see citing works
Jensen, J. B., Dollerup, O. L., Møller, A. B., Billeskov, T. B., Dalbram, E., Chubanava, S., Damgaard, M. V., Dellinger, R. W., Trošt, K., Moritz, T., et al. (2022) · JCI Insight · doi:10.1172/jci.insight.158314 · Cited by 39 · see citing works
Self-citationJagaraj, C. J., Parakh, S., & Atkin, J. D. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2020.581950 · Cited by 36 · see citing works
Molecular hallmarks of ageing in amyotrophic lateral sclerosis
Jagaraj, C. J., Shadfar, S., Kashani, S. A., Saravanabavan, S., Farzana, F., & Atkin, J. D. (2024) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-024-05164-9 · Cited by 33 · see citing works
Culprit or Bystander: Defective Mitophagy in Alzheimer’s Disease
Xie, C., Aman, Y., Adriaanse, B. A., Cader, M. Z., Plun‐Favreau, H., Xiao, J., & Fang, E. F. (2020) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2019.00391 · Cited by 29 · see citing works
NAD+ Metabolism and Diseases with Motor Dysfunction
Lundt, S., & Ding, S. (2021) · Genes · doi:10.3390/genes12111776 · Cited by 27 · see citing works
Luo, X., Kang, X., & Schönhuth, A. (2023) · Nature Machine Intelligence · doi:10.1038/s42256-022-00604-2 · Cited by 27 · see citing works
Casani-Cubel, J., Benlloch, M., Sanchis-Sanchis, C. E., Marín, R., Lajara-Romance, J. M., & Ortí, J. E. D. L. R. (2021) · Metabolites · doi:10.3390/metabo11020120 · Cited by 29 · see citing works
Self-citationChanneling Nicotinamide Phosphoribosyltransferase (NAMPT) to Address Life and Death
Reddy, V. G., Krider, I. S., Alves, E. T. M., Courey, J. M., Laham, M. S., & Thatcher, G. R. J. (2024) · Journal of Medicinal Chemistry · doi:10.1021/acs.jmedchem.3c02112 · Cited by 27 · see citing works
Zhang, X., Zhang, Y., Sun, A., & Ge, J. (2021) · Genes & Diseases · doi:10.1016/j.gendis.2021.04.001 · Cited by 23 · see citing works
NAD augmentation as a disease-modifying strategy for neurodegeneration
Dölle, C., & Tzoulis, C. (2025) · Trends in Endocrinology and Metabolism · doi:10.1016/j.tem.2025.03.013 · Cited by 24 · see citing works
NAD+ Precursors and Antioxidants for the Treatment of Amyotrophic Lateral Sclerosis
Obrador, E., Palmer, R. S., López‐Blanch, R., Dellinger, R. W., & Estrela, J. M. (2021) · Biomedicines · doi:10.3390/biomedicines9081000 · Cited by 22 · see citing works
Self-citationThe Diverse Roles of Reactive Astrocytes in the Pathogenesis of Amyotrophic Lateral Sclerosis
Yang, K., Liu, Y., & Zhang, M. (2024) · Brain Sciences · doi:10.3390/brainsci14020158 · Cited by 26 · see citing works
Molecular mechanisms and therapeutic strategies for neuromuscular diseases
Zambon, A. A., Falzone, Y. M., Bolino, A., & Previtali, S. C. (2024) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-024-05229-9 · Cited by 20 · see citing works
Poor Bone Quality in Patients With Amyotrophic Lateral Sclerosis
Caplliure-Llopis, J., Escrivá, D., Benlloch, M., de la Rubia Ortí, J. E., Estrela, J. M., & Barrios, C. (2020) · Frontiers in Neurology · doi:10.3389/fneur.2020.599216 · Cited by 17 · see citing works
Self-citationHosaka, T., Tsuji, H., & Tamaoka, A. (2021) · Processes · doi:10.3390/pr9091667 · Cited by 19 · see citing works
Li, A., Li, S., Wang, J., Chen, Y., & Lü, M. (2022) · Crystal Growth & Design · doi:10.1021/acs.cgd.2c01073 · Cited by 15 · see citing works
Fels, J. A., Casalena, G., Konràd, C., Holmes, H. E., Dellinger, R. W., & Manfredi, G. (2022) · Human Molecular Genetics · doi:10.1093/hmg/ddac118 · Cited by 15 · see citing works
Self-citationTherapeutic Targets in Amyotrophic Lateral Sclerosis: Focus on Ion Channels and Skeletal Muscle
Tarantino, N., Canfora, I., Camerino, G. M., & Pierno, S. (2022) · Cells · doi:10.3390/cells11030415 · Cited by 14 · see citing works
Exploring the Role of Metabolic Hormones in Amyotrophic Lateral Sclerosis
Moțățăianu, A., Mănescu, I. B., Şerban, G. M., Bărcuţean, L., Ion, V., Bălașa, R., & Andone, S. R. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25105059 · Cited by 13 · see citing works
Zemel, M. B. (2020) · Journal of Medicinal Food · doi:10.1089/jmf.2020.0105 · Cited by 12 · see citing works
Metabolomics as a Crucial Tool to Develop New Therapeutic Strategies for Neurodegenerative Diseases
Lanznaster, D., Dingeo, G., Samey, R. A., Emond, P., & Blasco, H. (2022) · Metabolites · doi:10.3390/metabo12090864 · Cited by 12 · see citing works
Emerging clinical investigational drugs for the treatment of amyotrophic lateral sclerosis
Martínez‐González, L., & Martı́nez, A. (2023) · Expert Opinion on Investigational Drugs · doi:10.1080/13543784.2023.2178416 · Cited by 12 · see citing works
Nicotinamide Adenine Dinucleotide (NAD + )-Dependent Signaling in Neurological Disorders
Bresque, M., Esteve, D., Pehar, M., & Vargas, M. R. (2023) · Antioxidants and Redox Signaling · doi:10.1089/ars.2023.0241 · Cited by 12 · see citing works
Lactate: A Theranostic Biomarker for Metabolic Psychiatry?
Caddye, E., Pineau, J., Reyniers, J., Ronen, I., & Colasanti, A. (2023) · Antioxidants · doi:10.3390/antiox12091656 · Cited by 11 · see citing works
Hong, Y., Zhang, Z., Yangzom, T., Chen, A., Lundberg, B. C., Fang, E. F., Siller, R., Sullivan, G. J., Zeman, J., Tzoulis, C., et al. (2024) · International Journal of Biological Sciences · doi:10.7150/ijbs.91624 · Cited by 11 · see citing works
The Scientific and Therapeutic Rationale for Off‐Label Treatments in Amyotrophic Lateral Sclerosis
Bedlack, R., Li, X., Evangelista, B., Panzetta, M. E., Kwan, J. Y., Gittings, L. M., & Sattler, R. (2024) · Annals of Neurology · doi:10.1002/ana.27126 · Cited by 9 · see citing works
Potential Therapeutic Interventions Targeting NAD+ Metabolism for ALS
Lundt, S., & Ding, S. (2024) · Cells · doi:10.3390/cells13171509 · Cited by 9 · see citing works
Ahmed, N. A., Baker, M. R., & Bashford, J. A. (2022) · Clinical Neurophysiology · doi:10.1016/j.clinph.2022.02.020 · Cited by 9 · see citing works
López‐Blanch, R., Palmer, R. S., Oriol‐Caballo, M., Moreno-Murciano, P., Dellinger, R. W., Estrela, J. M., & Obrador, E. (2023) · Neurotherapeutics · doi:10.1016/j.neurot.2023.10.011 · Cited by 5 · see citing works
Self-citationDrug Combination to Slow Down the Progression of Amyotrophic Lateral Sclerosis
Torres-Rico, M., Marín-Rodríguez, B., Arasmou-Idrovo, M. S., Garcı́a, A. G., & Pascual-Guerra, J. (2025) · Current Treatment Options in Neurology · doi:10.1007/s11940-025-00835-9 · Cited by 4 · see citing works
Hamilton, H. L., Akther, M., Anis, S., Colwell, C. B., Vargas, M. R., & Pehar, M. (2024) · Antioxidants and Redox Signaling · doi:10.1089/ars.2023.0360 · Cited by 4 · see citing works
Machine Learning-Based ALS Diagnosis Using Gene Expression Data
Vu, D.-L., & Le, H.-C. (2023) · RIVF International Conference on Computing and Communication Technologies (RIVF) · doi:10.1109/rivf60135.2023.10471816 · Cited by 4 · see citing works
Regulation of Stem Cell Function by NAD +
Feng, Y., Qiu, H., & Chen, D. (2025) · Physiology · doi:10.1152/physiol.00052.2024 · Cited by 3 · see citing works
Bushana, P. N., Schmidt, M. A., Rempe, M. J., Sorg, B. A., & Wisor, J. P. (2023) · SLEEP Advances · doi:10.1093/sleepadvances/zpad044 · Cited by 3 · see citing works
Amyotrofik Lateral Skleroz Patofizyolojisi ve Tedavi Yaklaşımları
YILDIRIM, Z., TOKTAŞ, D. N., DEMİR, Ö., Gül, Z., & Utsukarçi, B. Ş. (2023) · Hacettepe University Journal of the Faculty of Pharmacy · doi:10.52794/hujpharm.1064372 · Cited by 3 · see citing works
Clinical evidence for the use of NAD+ precursors to slow aging
Khatri, S., Abdellatif, M., & Sedej, S. (2025) · Geromedicine · doi:10.70401/geromedicine.2025.0008 · Cited by 3 · see citing works
Juan, M. E., Verdaguer, E., Prohens, R., Ettcheto, M., Auladell, C., Camins, A., & Olloquequi, J. (2026) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2026.178620 · Cited by 2 · see citing works
Johanson, G. A. S., Tysnes, O., & Bjerknes, T. L. (2022) · Neurology Research International · doi:10.1155/2022/1789946 · Cited by 2 · see citing works
Autonomic nervous system and mediating role of respiratory function in patients with ALS
de Bernardo, N., Ortí, J. E. D. L. R., Villarón-Casales, C., Privado, J., Maset-Roig, R., Cañabate, M., Sancho-Cantus, D., Sanz, I. O., Fernández, R. F., Proaño, B., et al. (2025) · Scientific Reports · doi:10.1038/s41598-025-94844-y · Cited by 2 · see citing works
Self-citationNeurodegenerative Diseases and the Gut Microbiota
Cabré, S., O’Riordan, K. J., & Cryan, J. F. (2022) · Progress in inflammation research · doi:10.1007/978-3-030-91051-8_11 · Cited by 2 · see citing works
Lin, Z., Ibrahim, A. N., Brown, R. H., & Sreedharan, J. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-19176-3.00034-0 · Cited by 2 · see citing works
Carrera‐Juliá, S., Catarina-Moreira, A., Santos, C. A., Fonseca, J., & Drehmer, E. (2021) · Revista Española de Nutrición Humana y Dietética · doi:10.14306/renhyd.25.4.1308 · Cited by 1 · see citing works
Self-citationAdvanced Gene Selection Methods for Machine Learning-Based Amyotrophic Lateral Sclerosis Diagnosis
Nguyen, H.-L., Dang, D., & Le, H.-C. (2025) · RIVF International Conference on Computing and Communication Technologies (RIVF) · doi:10.1109/rivf68649.2025.11365068 · Cited by 1 · see citing works
Microbiota as a regulator of brain vulnerability across lifespan and disease contexts
Santerre, M., Shcherbik, N., & Sawaya, B. E. (2026) · Journal of Neuroinflammation · doi:10.1186/s12974-026-03824-0 · Cited by 1 · see citing works
Exploiting Machine Learning And Gene Expression Analysis in Amyotrophic Lateral Sclerosis Diagnosis
Nguyen, H.-L., Vu, D.-L., & Le, H.-C. (2024) · International Conference on Communications and Electronics (ICCE) · doi:10.1109/icce62051.2024.10634725 · Cited by 1 · see citing works
ALSUntangled no. 49: resveratrol
THE ALSUNTANGLED GROUP (2019) · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · doi:10.1080/21678421.2019.1593596 · Cited by 1 · see citing works
Introduction to Novel Motor Neuron Disease
Sibat, H. F., & Valdés, L. D. F. I. (2020) · IntechOpen eBooks · doi:10.5772/intechopen.91921
New therapeutics to modulate mitochondrial energy metabolism in neurodegenerative disorders
Orsucci, D., Ienco, E. C., Siciliano, G., & Mancuso, M. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-819621-2.00023-1
Mitochondria and Energy Metabolism in the Pathogenesis and Therapy of Amyotrophic Lateral Sclerosis
Konràd, C., & Manfredi, G. (2023) · Oxford University Press eBooks · doi:10.1093/oxfordhb/9780190932183.013.14
Cornélissen, G., Gubin, D. G., & Otsuka, K. (2024) · Royal Society of Chemistry eBooks · doi:10.1039/bk9781839167553-00455
Current Understanding of ALS Onset, Progression and TreatmentsPreprint
Rossmiller, B. P., & Bagheri, M. (2024) · doi:10.14293/pr2199.001277.v1
Prajapati, J. L., Dhurandhar, Y., Singh, A. K., Gupta, D. K., Baghel, V. S., Kushwaha, U., & Namdeo, K. P. (2025) · Expert Opinion on Drug Delivery · doi:10.1080/17425247.2025.2489558
Avances en la investigación sobre longevidad: focos actuales y perspectivas futuras
Monje, L. F. C. (2024) · Medicina · doi:10.56050/01205498.2337
Bloom, A. J., Mao, X., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Figshare · doi:10.6084/m9.figshare.17982739
Bloom, A. J., Mao, X., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Figshare · doi:10.6084/m9.figshare.17982727
Bloom, A. J., Mao, X., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Figshare · doi:10.6084/m9.figshare.17982736
Bloom, A. J., Mao, X., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Figshare · doi:10.6084/m9.figshare.17982742
Bloom, A. J., Mao, X., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Figshare · doi:10.6084/m9.figshare.17982721
Bloom, A. J., Mao, X., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Figshare · doi:10.6084/m9.figshare.17982733
Bloom, A. J., Mao, X., Strickland, A. M., Sasaki, Y., Milbrandt, J., & DiAntonio, A. (2022) · Figshare · doi:10.6084/m9.figshare.17982730
Zhou, S., Li, X., Jiao, Y., & Wu, J. (2026) · Frontiers in Neurology · doi:10.3389/fneur.2026.1754716
BindingDB (2025) · UC San Diego · doi:10.7270/q25x2fk7
- Authors
- de la Rubia, J. E., Drehmer, E., Platero, J. L., Benlloch, M., Caplliure-Llopis, J., Villaron-Casales, C., de Bernardo, N., AlarcÓn, J., Fuente, C., Carrera, S., Sancho, D., GarcÍa-Pardo, P., Pascual, R., JuÁrez, M., Cuerda-Ballester, M., Forner, A., Sancho-Castillo, S., Barrios, C., Obrador, E., Marchio, P., Salvador, R., Holmes, H. E., Dellinger, R. W., Guarente, L., & Estrela, J. M.
- Affiliations
- Department of Nursing, Catholic University San Vicente Mártir, Valencia, Spain,
- Valencia Catholic University Saint Vincent Martyr (ES)
- Primary Care Service, University Hospital La Ribera, Alcira, Spain,
- Department of Physiotherapy, European University of Valencia, Valencia, Spain,
- Department of Medicine, University Jaume I, Castellón, Spain,
- Hospital General Universitario De Valencia (ES)
- Clínica Artes & Timefit, Valencia, Spain,
- Universitat de València (ES)
- Virgen del Consuelo Hospital, Valencia, Spain,
- Department of Physiology, University of Valencia, Valencia, Spain,
- Elysium Health, Inc. (United States) (US)
- Corresponding author
- Estrela, José M.
- ORCID
- Venue
- Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
- Publisher
- Informa UK Limited
- Published
- 2019-01-02
- Volume
- 20
- Issue
- 1-2
- Pages
- 115-122
- ISSN
- 2167-8421 (ISSN-L)
- 2167-9223
- OpenAlex
- W2911693028
- Cited by
- 92
- References
- 30
- Citations by year
- 2026: 3 · 2025: 9 · 2024: 16 · 2023: 12 · 2022: 18 · 2021: 13 · 2020: 19 · 2019: 2
- FWCI
- 8.77
- Type
- journal article
- Language
- en
- Open access
- green
- hdl.handle.net
- Licence
- cc-by-nc-nd
- Locations
- Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration (publishedVersion)
- PubMed (publishedVersion)
- RIUCV (Universidad Católica de Valencia San Vicente Martír) (submittedVersion, cc-by-nc-nd)
- ABACUS. Repositorio de Producción Científica (Universidad Europea) (submittedVersion)
- Hispana (submittedVersion, cc-by-nc-nd)
- Repositori UJI (Universitat Jaume I) (submittedVersion, public-domain)
- Repository of Digital Objects for Teaching Research and Culture (University of Valencia) (submittedVersion, cc-by-nc)
- Funders
- Catholic University San Vicente Mártir (2017-216-001)
- University of Valencia (OTR2017-18255INVES)
- Topics
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Keywords
- amyotrophic lateral sclerosis, disease-modifying therapy, drug efficacy, tolerability, ALSFRS-R, forced vital capacity, lung function, muscle strength, disease progression, neurodegenerative diseases
- MeSH terms
- Aged, Amyotrophic Lateral Sclerosis, Double-Blind Method, Drug Combinations, Electromyography, Female, Humans, Male, Middle Aged, Niacinamide, Pilot Projects, Ribonucleosides, Stilbenes, Vital Capacity, Treatment Outcome, Disease Progression, Muscle Strength
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
