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Review

Targeting Ferroptosis in Rare Neurological Disorders Including Pediatric Conditions: Innovations and Therapeutic Challenges

by
Ahmed D. Alatawi
1,
Krishnaraju Venkatesan
2,*,
Khalid Asseri
2,
Premalatha Paulsamy
3,
Saleh F. Alqifari
4,
Rehab Ahmed
5,
Mathar Mohideen Nagoor Thangam
6,
Nizar Sirag
5,
Absar A. Qureshi
2,
Hala Ahmed Elsayes
7,8,
Zeinab Faried Bahgat
9,10,
Nesren S. M. Bahnsawy
11,12,
Kousalya Prabahar
4 and
Basma Mahmoud Abd Elhamid Dawood
13,14
1
Department of Clinical Pharmacy, College of Pharmacy, Jouf University, Sakaka 72388, Saudi Arabia
2
Department of Pharmacology, College of Pharmacy, King Khalid University, Abha 62521, Saudi Arabia
3
College of Nursing, Mahalah Branch for Girls, King Khalid University, Abha 62521, Saudi Arabia
4
Department of Pharmacy Practice, Faculty of Pharmacy, University of Tabuk, Tabuk 71491, Saudi Arabia
5
Department of Natural Products and Alternative Medicine, Faculty of Pharmacy, University of Tabuk, Tabuk 71491, Saudi Arabia
6
Department of Medical Surgical Nursing, Faculty of Nursing, University of Tabuk, Tabuk 71491, Saudi Arabia
7
Department of Psychiatric and Mental Health Nursing, King Abdulaziz University, Jeddah 21589, Saudi Arabia
8
Department of Psychiatric and Mental Health, Faculty of Nursing, Tanta University, Tanta 31527, Egypt
9
Department of Medical-Surgical Nursing, Faculty of Nursing, Tanta University, Tanta 31527, Egypt
10
Department of Medical-Surgical Nursing, College of Nursing, King Saud Bin Abdul Aziz University for Health Sciences (KSAU-HS), King Abdullah International Medical Research Center, Al-Ahsa 31982, Saudi Arabia
11
Department of Pediatric Nursing, College of Nursing, King Saud Bin Abdul Aziz University for Health Sciences (KSAU-HS), King Abdullah International Medical Research Center, Ministry of National Guard Health Affairs, Riyadh 11481, Saudi Arabia
12
Department of Pediatric Nursing, Faculty of Nursing, Cairo University, Giza 12613, Egypt
13
Department of Pediatric Nursing, Faculty of Nursing, Tanta University, Tanta 31527, Egypt
14
Department of Pediatric Nursing, College of Nursing, King Saud Bin Abdul Aziz University for Health Sciences (KSAU-HS), King Abdullah International Medical Research Center, Al-Ahsa 31982, Saudi Arabia
 
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*
Author to whom correspondence should be addressed.
Biomedicines 2025, 13(2), 265; https://doi.org/10.3390/biomedicines13020265
Submission received: 31 December 2024 / Revised: 9 January 2025 / Accepted: 16 January 2025 / Published: 22 January 2025
(This article belongs to the Special Issue Advancements in Brain Diagnosis and Treatment: From Insights to Innovations)
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Abstract

Ferroptosis, characterized by iron dependency and lipid peroxidation, has emerged as a key mechanism underlying neurodegeneration in rare neurological disorders. These conditions, often marked by significant therapeutic gaps and high unmet medical needs, present unique challenges for intervention development. This review examines the involvement of ferroptosis in rare neurological disease pathogenesis, focusing on its role in oxidative damage and neuronal dysfunction. We explore recent pharmacological advancements, including iron chelators, lipid peroxidation blockers, and antioxidant-based strategies, designed to target ferroptosis. While these approaches show promise, challenges such as disease heterogeneity, limited diagnostic tools, and small patient cohorts hinder progress. Furthermore, we discuss the translational and regulatory barriers to implementing ferroptosis-based therapies in clinical practice. By addressing these obstacles and fostering innovative solutions, this review underscores the potential of ferroptosis-targeting strategies to revolutionize treatment paradigms for rare neurological disorders.
Keywords: ferroptosis; rare neurological disorders; lipid peroxidation; iron metabolism ferroptosis; rare neurological disorders; lipid peroxidation; iron metabolism

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MDPI and ACS Style

Alatawi, A.D.; Venkatesan, K.; Asseri, K.; Paulsamy, P.; Alqifari, S.F.; Ahmed, R.; Nagoor Thangam, M.M.; Sirag, N.; Qureshi, A.A.; Elsayes, H.A.; et al. Targeting Ferroptosis in Rare Neurological Disorders Including Pediatric Conditions: Innovations and Therapeutic Challenges. Biomedicines 2025, 13, 265. https://doi.org/10.3390/biomedicines13020265

AMA Style

Alatawi AD, Venkatesan K, Asseri K, Paulsamy P, Alqifari SF, Ahmed R, Nagoor Thangam MM, Sirag N, Qureshi AA, Elsayes HA, et al. Targeting Ferroptosis in Rare Neurological Disorders Including Pediatric Conditions: Innovations and Therapeutic Challenges. Biomedicines. 2025; 13(2):265. https://doi.org/10.3390/biomedicines13020265

Chicago/Turabian Style

Alatawi, Ahmed D., Krishnaraju Venkatesan, Khalid Asseri, Premalatha Paulsamy, Saleh F. Alqifari, Rehab Ahmed, Mathar Mohideen Nagoor Thangam, Nizar Sirag, Absar A. Qureshi, Hala Ahmed Elsayes, and et al. 2025. "Targeting Ferroptosis in Rare Neurological Disorders Including Pediatric Conditions: Innovations and Therapeutic Challenges" Biomedicines 13, no. 2: 265. https://doi.org/10.3390/biomedicines13020265

APA Style

Alatawi, A. D., Venkatesan, K., Asseri, K., Paulsamy, P., Alqifari, S. F., Ahmed, R., Nagoor Thangam, M. M., Sirag, N., Qureshi, A. A., Elsayes, H. A., Faried Bahgat, Z., Bahnsawy, N. S. M., Prabahar, K., & Dawood, B. M. A. E. (2025). Targeting Ferroptosis in Rare Neurological Disorders Including Pediatric Conditions: Innovations and Therapeutic Challenges. Biomedicines, 13(2), 265. https://doi.org/10.3390/biomedicines13020265

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