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Most Advanced Therapeutic Agents Redefining Neurological Health

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Clinical Neurology".

Deadline for manuscript submissions: closed (15 April 2026) | Viewed by 796

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Guest Editor
Neuroscience and Behavior Program, Wesleyan University, Middletown, CT 06459, USA
Interests: neurodegeneration; neuroimmunomodulation; neuroscience and behavior
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Recent clinical trials have established several novel therapeutics as practice-changing interventions in neurology. Regarding the treatment of Alzheimer's disease, anti-amyloid monoclonal antibodies (lecanemab, donanemab) now demonstrate the statistically significant slowing of cognitive decline (CDR-SB 27–35%) in phase 3 trials when initiated at early disease stages. Parkinson's therapeutics now include targeted agents like safinamide (MAO-B/glutamate modulation), showing improved motor fluctuations in extension studies, while subcutaneous apomorphine infusion provides effective rescue therapy. In MS, CD20-depleting therapies (ocrelizumab, ofatumumab) achieve 85–90% relapse reduction with manageable infection risks. Acute neuroprotection is now achievable with tenecteplase (0.4 mg/kg) for large-vessel occlusion strokes, showing superior reperfusion to alteplase in recent RCTs. For SMA, onasemnogene abeparvovec produces 90% survival without ventilation at 24 months when administered presymptomatically. These advances mandate updated treatment algorithms, emphasizing (1) earlier biomarker-guided intervention, (2) combination approaches, and (3) rigorous monitoring for ARIA (amyloid therapies) and IRIS (immune reconstitution) effects.

Dr. Clarissa Fantin Cavarsan
Guest Editor

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Keywords

  • disease-modifying therapy
  • biomarker-guided treatment
  • gene therapy
  • neuroprotection
  • clinical trials
  • neurological disorders
  • precision neurology

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Published Papers (1 paper)

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15 pages, 1569 KB  
Systematic Review
Alternative Treatment of the Resistant-to-Treatment Tourette Syndrome—A Systematic Review
by Agata Czech, Magdalena Czarnecka, Olga Grodzka, Piotr Chądzyński and Izabela Domitrz
J. Clin. Med. 2026, 15(9), 3393; https://doi.org/10.3390/jcm15093393 - 29 Apr 2026
Viewed by 530
Abstract
Background: Tourette syndrome (TS) is a chronic neurodevelopmental disorder with a significant rate of patients remaining refractory to standard treatments. Refractory TS is defined as the persistence of clinically significant tics causing functional impairment despite adequate trials of both behavioral therapy and at [...] Read more.
Background: Tourette syndrome (TS) is a chronic neurodevelopmental disorder with a significant rate of patients remaining refractory to standard treatments. Refractory TS is defined as the persistence of clinically significant tics causing functional impairment despite adequate trials of both behavioral therapy and at least two generally accepted pharmacological treatments from different classes, administered at appropriate doses and durations. This systematic review synthesizes the current evidence on alternative pharmacological and non-pharmacological interventions for treatment-resistant TS. Methods: The review was conducted in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guidelines. PubMed, Embase, and Scopus were searched for studies published from 2010 onward. Eligible publications included randomized and non-randomized clinical studies, and case reports evaluating alternative interventions in refractory TS, excluding deep brain stimulation. The primary outcome measures included validated scales assessing tic severity and functional impairment, with available data demonstrating reductions in tic severity, including clinically meaningful improvements reported in several studies. Thirteen studies met the inclusion criteria, comprising five clinical studies (including three randomized trials) and eight case reports. Heterogeneity across studies was primarily driven by differences in study design, patient populations, and variability in intervention types, protocols, and outcome assessments. Results: Investigated interventions included cannabinoids, valproic acid, deep transcranial magnetic stimulation, neurofeedback, biofeedback, electroconvulsive therapy, and ablative neurosurgical procedures. Cannabinoid-based treatments showed potential reductions in tic severity; however, results were inconsistent and often not statistically significant, with evidence largely derived from small or uncontrolled studies. Evidence for non-pharmacological approaches was limited and largely derived from individual cases, with some modalities showing potential benefit in specific subgroups, such as patients with comorbid obsessive-compulsive disorder. Conclusions: Overall, alternative interventions may offer therapeutic value for selected individuals with treatment-resistant TS, with the greatest data regarding cannabinoid use; however, the current evidence base remains heterogeneous and methodologically constrained. Deep transcranial magnetic stimulation might be beneficial in a specific patient population. Larger, well-controlled studies are required to clarify efficacy, safety, and treatment response. Full article
(This article belongs to the Special Issue Most Advanced Therapeutic Agents Redefining Neurological Health)
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