Inner ear disorders, including sensorineural hearing loss, Meniere’s disease, and vestibular neuritis, are prevalent conditions that significantly impact the quality of life. Despite their high incidence, the underlying pathophysiology of these disorders remains elusive, and current treatment options are often inadequate. Emerging evidence suggests that pericytes, a type of vascular mural cell specialized to maintain the integrity and function of the microvasculature, may play a crucial role in the development and progression of inner ear disorders. The pericytes are present in the microvasculature of both the cochlea and the vestibular system, where they regulate blood flow, maintain the blood–labyrinth barrier, facilitate angiogenesis, and provide trophic support to neurons. Understanding their role in inner ear disorders may provide valuable insights into the pathophysiology of these conditions and lead to the development of novel diagnostic and therapeutic strategies, improving the standard of living. This comprehensive review aims to provide a detailed overview of the role of pericytes in inner ear disorders, highlighting the anatomy and physiology in the microvasculature, and analyzing the mechanisms that contribute to the development of the disorders. Furthermore, we explore the potential pericyte-targeted therapies, including antioxidant, anti-inflammatory, and angiogenic approaches, as well as gene therapy strategies.

Background/Objectives: Rare diseases are a wide and heterogeneous group of multisystem life-threatening or chronically debilitating clinical conditions with reduced life expectancy and a relevant mortality rate in childhood. Some of these disorders have typical neurological symptoms, presenting from birth to adulthood. Dietary patterns and nutritional compounds play key roles in the onset and progression of neurological disorders, and the impact of alimentary needs must be enlightened especially in rare neurological diseases. This work aims to collect the in vitro, in vivo, and clinical evidence on the effects of diet and of nutrient intake on some rare neurological disorders, including some genetic diseases, and rare brain tumors. Herein, those aspects are critically linked to the genetic, biological, biochemical, and pathophysiological hallmarks typical of each disorder. Methods: By searching the major web-based databases (PubMed, Web of Science Core Collection, DynaMed, and Clinicaltrials.gov), we try to sum up and improve our understanding of the emerging role of nutrition as both first-line therapy and risk factors in rare neurological diseases. Results: In line with the increasing number of consensus opinions suggesting that nutrients should receive the same attention as pharmacological treatments, the results of this work pointed out that a standard dietary recommendation in a specific rare disease is often limited by the heterogeneity of occurrent genetic mutations and by the variability of pathophysiological manifestation. Conclusions: In conclusion, we hope that the knowledge gaps identified here may inspire further research for a better evaluation of molecular mechanisms and long-term effects.

Background/Objectives: Medulloblastoma is a rare tumor that represents almost two-thirds of all embryonal pediatric brain tumor cases. Current treatments, including surgery, radiation, and chemotherapy, are often associated with adverse effects, such as toxicity, resistance, and lack of specificity. According to multiple bulk and single-cell omics-based approaches, it is now clear that each molecular subgroup of medulloblastoma possesses intrinsic genetic and molecular features that could drive the definition of distinct therapeutic targets, and of markers that have the potential to improve diagnosis. Nanomedicine offers a promising approach to overcome these challenges through precision-targeted therapies and theranostic platforms that merge diagnosis and treatment. This review explores the role of nanomedicine in medulloblastoma. Here, possible theranostic nanoplatforms combining targeted drug delivery and simultaneous imaging are reviewed, highlighting their potential as tools for personalized medicine. Methods: We performed a chronological analysis of the literature by using the major web-based research platforms, focusing on molecular targets, and the potential application of nanomedicine to overcome conventional treatment limitations. Results: Advances in nanoparticle-based drug delivery systems enable selective targeting of key molecular pathways, improving therapeutic efficacy while minimizing off-target effects. Additionally, nanotechnology-based imaging agents, including MRI contrast agents and fluorescent probes, improve diagnostic accuracy and treatment monitoring. Despite these advantages, some significant challenges remain, including overcoming the blood–brain barrier, ensuring biocompatibility, and addressing regulatory pathways for clinical translation. Conclusions: In conclusion, we sought to identify the current knowledge on the topic and hope to inspire future research to obtain new nanoplatforms for personalized medicine.

Background/Objectives: Difficult-to-treat epileptic syndromes include conditions typically emerging in the first years of life and are characterized by a high rate of drug refractoriness. This study aimed to better define the safety profile of drugs used as adjunctive therapies for seizures associated with these syndromes using real-world pharmacovigilance data. Methods: We retrospectively analyzed the publicly available data regarding Individual Case Safety Reports (ICSRs), presenting stiripentol, cannabidiol, or fenfluramine as suspected drugs, reported on the Eudravigilance database until the third quarter of 2024. Data were evaluated with descriptive analyses and then with disproportionality measures, including the reporting odds ratio. Results: A total of 5986 ICSRs met the inclusion criteria (71.6% from cannabidiol, 14.5% fenfluramine, and 13.9% stiripentol). Significantly higher probabilities of reporting Cardiac disorders, Vascular disorders, and Respiratory, thoracic, and mediastinal disorders were observed with fenfluramine. Cannabidiol was associated with Product issues, whereas stiripentol was associated with injury, poisoning, procedural complications, Metabolism and nutrition disorders, and Blood and lymphatic system disorders. Conclusions: Our analysis did not highlight new and unexpected serious safety signals but confirmed the need to strictly monitor patients for the risk of adverse events. However, further prospective studies are required to better clarify the safety profile of these drugs in order to optimize their use.