Efficacy and Safety of Fenfluramine in Epilepsy: A Systematic Review and Meta-analysis
ConclusionFenfluramine reduces the frequency of seizures in patients with DS and LGS. Decreased appetite, diarrhoea, fatigue, and weight loss are non-cardiovascular AEs associated with FFA. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 28, 2023 Category: Neurology Source Type: research

Discrete Choice Experiment to Understand Japanese Patients ’ and Physicians’ Preferences for Preventive Treatments for Migraine
ConclusionMany patients and physicians preferred CGRP mAb auto-injectors over non-CGRP oral medications and preferred a treatment profile similar to galcanezumab. Our results may encourage physicians in Japan to consider patient preferences when recommending migraine preventive treatments. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 27, 2023 Category: Neurology Source Type: research

Characterization of Disease Severity and Stability in NMOSD: A Global Clinical Record Review with Patient Interviews
ConclusionA clear consensus on how to assess relapse severity and disease stability is needed to ensure that patients receive appropriate and timely treatment. In the future, clinical measures should be combined with patient-focused assessments. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 24, 2023 Category: Neurology Source Type: research

Understanding Treatment Decisions in Neuromyelitis Optica Spectrum Disorder: A Global Clinical Record Review with Patient Interviews
ConclusionMisdiagnosis of NMOSD appears to be common and is associated with a delay in initiation of maintenance therapy, with decisions influenced by relapse severity. Further real-world studies assessing relapse severity in treatment initiation/switch are required to revise NMOSD treatment recommendations. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 24, 2023 Category: Neurology Source Type: research

Correction to: Multiple Sclerosis and Neuromyelitis Optica Spectrum Disorder: Onset Following Acute COVID-19 Infection, a Case Series
(Source: Neurology and Therapy)
Source: Neurology and Therapy - February 23, 2023 Category: Neurology Source Type: research

Pridopidine Does Not Significantly Prolong the QTc Interval at the Clinically Relevant Therapeutic Dose
ConclusionsAt the 45  mg bid therapeutic dose, pridopidine demonstrates a favorable cardiac safety profile, with an effect on the QTc interval that is below the level of concern and not clinically relevant.Trial RegistrationPRIDE-HD (TV7820-CNS-20002) trial registration: ClinicalTrials.gov identifier, NCT02006472, EudraCT 2013-001888-23; HART (ACR16C009) trial registration: ClinicalTrials.gov identifier, NCT00724048; MermaiHD (ACR16C008) trial registration: ClinicalTrials.gov identifier, NCT00665223, EudraCT No. 2007-004988-22. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 22, 2023 Category: Neurology Source Type: research

Two Perspectives on Dravet Syndrome: Viewpoints from the Clinician and the Caregiver
AbstractDravet syndrome (DS) is a severe genetic epilepsy characterized by early-life onset, seizures, and neurodevelopmental delays that have major impacts on affected children. DS is an incurable condition that requires a lifelong multidisciplinary approach involving both clinical and caregiver support. A better understanding of the multiple perspectives involved in the care of patients is necessary for supporting the diagnosis, management, and treatment of DS. Here we describe the personal experiences of a caregiver and a clinician facing the challenges of diagnosing and treating a patient throughout the three phases of...
Source: Neurology and Therapy - February 16, 2023 Category: Neurology Source Type: research

COVID-19 Vaccine Response in People with Multiple Sclerosis Treated with Dimethyl Fumarate, Diroximel Fumarate, Natalizumab, Ocrelizumab, or Interferon Beta Therapy
ConclusionsThese results suggest that humoral response to mRNA-1273 COVID-19 vaccine is preserved and similar in PwMS treated with natalizumab, fumarate, and interferon  beta, but muted with ocrelizumab. All DMTs had preserved T cell response, including the ocrelizumab cohort, which also had a greater risk of vaccine-related side effects. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 16, 2023 Category: Neurology Source Type: research

Technical Review of Clinical Outcomes Assessments Across the Continuum of Alzheimer's Disease
ConclusionWhile many frequently used COAs in AD capture some concepts identified as important to AD populations and their care partners, overlap between any single measure and the concepts that matter to people affected by AD is limited. The highest singly matched COA reflects fewer than half (45%) of WMM concepts. Use of multiple COAs expands coverage of meaningful concepts. Future research should explore the content validity of AD COAs planned for AD trials based on further confirmation of the ecological validity of the WMM items. This research should inform development and use of core outcome sets that capture WMM items...
Source: Neurology and Therapy - February 15, 2023 Category: Neurology Source Type: research

Identification of Potential Serum Protein Biomarkers in Thymoma with Myasthenia Gravis After Docetaxel Treatment
ConclusionsOur study revealed that the serum proteins were differentially expressed after docetaxel treatment, suggesting their important role in patients with TMG, as well as the critical role of KRAS and SELP as biomarkers in evaluating the efficacy of docetaxel treatment. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 14, 2023 Category: Neurology Source Type: research

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study
ConclusionsThe safety and PD of risdiplam in patients who were previously treated were consistent with those of treatment-na ïve patients. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 13, 2023 Category: Neurology Source Type: research

Real-World Effectiveness of Natalizumab Extended Interval Dosing in a French Cohort
ConclusionThese results support the pertinence of using an EID strategy for RRMS patients treated with natalizumab.Clinical Trialsgov identifier (NCT04580381). (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 10, 2023 Category: Neurology Source Type: research

Assessing What Matters to People Affected by Alzheimer ’s Disease: A Quantitative Analysis
ConclusionConcepts of importance to individuals affected by AD go beyond the common understanding of “cognition” or “function” alone, reflecting a desire to maintain independence, overall physical and mental health, emotional well-being, and safety. Preservation of these attributes may be key to understanding whether interventions deliver clinically meaningful outcomes. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 10, 2023 Category: Neurology Source Type: research

Assisted Reproductive Techniques in Multiple Sclerosis: Recommendations from an Expert Panel
ConclusionsEarly preconception consultations are essential to individualising pregnancy management in women with MS, and an early, well-planned, spontaneous pregnancy should be the aim whenever possible. The management of women with MS and the desire for motherhood by multidisciplinary units is warranted to ensure appropriate guidance through the entire pregnancy. (Source: Neurology and Therapy)
Source: Neurology and Therapy - February 7, 2023 Category: Neurology Source Type: research

Unmet Needs in Preventive Treatment of Migraine
AbstractMigraine represents the most common cause of work disability in young women and the second one in the general population. Preventive treatment can reduce the frequency of attacks and their intensity, consequently improving the quality of life. Despite this, global health systems have shown important gaps in addressing optimal management of preventive therapy. Despite numerous adverse effects of traditional medications for migraine prevention being well known, these medications continue to be considered the standard of care for prophylaxis of this disease in many contexts. On the other hand, the widespread use of ca...
Source: Neurology and Therapy - February 4, 2023 Category: Neurology Source Type: research