Diseases

Background/Objectives: Cannabinoids are increasingly recognised for their therapeutic potential beyond well-established indications such as chronic pain, multiple sclerosis, and specific epileptic syndromes. Recent advances have highlighted their possible role in less-common or orphan diseases, opening new avenues for pharmaceutical research and clinical application. Methods: This review provides a critical synthesis of the most recent evidence (2020–2025), available in PubMed and Scopus, regarding the use of cannabinoids in conditions including refractory epilepsies beyond Dravet and Lennox–Gastaut syndromes, movement disorders such as dystonia and Tourette syndrome, rare dermatological diseases like epidermolysis bullosa, and emerging data in Crohn’s disease. Results: Negative outcomes, such as those reported in Fragile X syndrome trials, are also discussed as instructive examples of methodological and pharmacological challenges. Particular attention is given to the optimisation of pharmaceutical formulations and advanced separation technologies, including oromucosal sprays, transdermal gels, and novel nanocarrier systems, which aim to overcome issues of bioavailability and variability in patient response. Finally, safety concerns, regulatory aspects, and the need for robust clinical trials are addressed. Conclusions: Overall, cannabinoids represent a promising yet underexplored therapeutic option in rare and complex disorders, warranting further investigation supported by innovative pharmaceutical approaches.

Background/Objectives: Encephalitis and related acute encephalopathic syndromes represent severe neurological conditions with diverse etiologies and variable clinical outcomes. This study aimed to analyze nationwide hospitalization patterns for encephalitis-related diagnoses in Ecuador between 2018 and 2024. Methods: We used data from the Ecuadorian National Institute of Statistics and Census to estimate age-adjusted hospitalization and mortality rates according to ICD-10 codes. Binary and multinomial logistic regression models were employed to identify sociodemographic factors and diagnostic categories of encephalitis associated with hospitalization and in-hospital mortality. Results: A total of 1560 hospitalizations related to encephalitis-spectrum diagnoses were recorded, with an overall age-adjusted rate of 0.127 per 100,000 inhabitants and 6.0% in-hospital mortality. Unspecified encephalitis and encephalomyelitis were the most common diagnostic categories. Adolescents (10–19 years) were more frequently diagnosed with acute disseminated and bacterial meningoencephalitis, while patients aged ≥70 had higher odds of “other” encephalitis subtypes and the highest mortality risk (aOR = 0.265; 95% CI: 0.116–0.608). Indigenous individuals were more likely to be diagnosed with acute disseminated encephalitis, and Black individuals showed a higher risk for myelopathy associated with human T-cell lymphotropic virus type 1-associated myelopathy. Conclusions: Age and ethnicity significantly influence hospitalization due to encephalitis-related diagnoses in Ecuador. These findings provide epidemiological rates for a lower-middle–income country where the lack of precise diagnosis, age, and ethnicity contribute to the vulnerability of encephalitis.

Background: Multiple myeloma (MM) is a clonal plasma cell neoplasm representing the second most common hematological malignancy. The combination of daratumumab, lenalidomide and dexamethasone (D-Rd) was first approved by the EMA (European Medicines Agency) for the treatment of relapsed/refractory multiple myeloma (RRMM) patients, and was subsequently approved for first-line therapy, based on the results of POLLUX and MAIA trials, respectively. Methods: In this survey, we retrospectively collected data from 96 consecutive transplant-ineligible newly diagnosed multiple myeloma (TIE-NDMM) patients treated with the D-Rd combination. Results: The median age was 73 years; the median progression free survival (mPFS) and median overall survival (mOS) were not reached (NR); the overall response rate (ORR), defined as patients who obtained at least a partial response (PR), was 90%; 59% of patients achieved a very good partial response (VGPR) or better. A strong negative correlation was observed between treatment response and elevated beta-2-microglobulin levels. Conclusions: This study confirms the efficacy of the D-Rd combination as first-line therapy for TIE-NDMM patients, suggesting that achieving at least a PR—and particularly a VGPR—may represent a strong predictor of long-term remission and survival, even in the era of new combinations based on the use of quadruplets.