Patterns and predictors of therapeutic response to efgartigimod in acetylcholine receptor-antibody generalized myasthenia gravis subtypes
Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be life-threatening. However, the therapeutic response to efgartigimod among the acetylcholine receptor gMG (AChR-gMG) subtypes remains inconclusive.
CSF IL-6, GDF-15, GFAP and NfL levels in early Alzheimer disease: a pilot study
Despite their potential usefulness as biomarkers, no study has investigated the interactions between cerebrospinal fluid (CSF) changes of neurofilament light chain (NfL), glial fibrillary acidic protein (GFAP), growth differentiation factor 15 (GDF-15), transactive response DNA binding protein (TDP-43) and interleukin-6 (IL-6) and the core AD CSF biomarkers in the same cohort of AD patients.
Sustained-release oral dalfampridine appears to have no impact on upper extremity function in people with multiple sclerosis: a randomized controlled trial
Upper limb dysfunction is common in people with multiple sclerosis (pwMS), significantly affecting daily activities and quality of life. While dalfampridine has shown efficacy in improving gait in pwMS, its impact on upper extremity function remains unclear.
Resolution of bilateral temporal lobe epilepsy via minimally invasive stereoelectroencephalogram-guided asymmetric radiofrequency thermocoagulation: a case report
Bilateral temporal lobe epilepsy (TLE) is a complex form of epilepsy, characterized by seizures originating from both temporal lobes. Its intricate nature presents significant challenges for both medical and surgical treatment, often necessitating a multidisciplinary approach. While resective surgery is generally unsuitable for bilateral TLE, neuromodulation offers more of a remission-focused approach, which better preserves neuropsychological function. In this study, we presented the case of a 45-year-old female with a 16-years history of recurrent seizures, diagnosed as drug-resistant bilateral TLE. After a comprehensive multidisciplinary epilepsy evaluation-encompassing detailed patient history, neurological examination, scalp audiovisual electroencephalogram monitoring, high-resolution brain magnetic resonance imaging, neuropsychological testing, and cerebrospinal fluid analysis-stereoelectroencephalogram (SEEG) confirmed the diagnosis. The patient subsequently underwent asymmetric radiofrequency thermocoagulation guided by SEEG results, targeting bilateral temporal lobes. Despite experiencing transient psychiatric symptoms postprocedure, she achieved seizure freedom and showed improved neuropsychological function over a 3-year follow-up period. This case demonstrates that, with thorough evaluation, seizure freedom is attainable in bilateral TLE patients, even when bilateral injury is present, without significant impairment to neuropsychological function.
A multi-country cohort database study to assess pregnancy and infant outcomes after potential maternal or paternal exposure to cladribine tablets in the treatment of multiple sclerosis: the CLEAR study methods and status update
Cladribine tablets are contraindicated during pregnancy; therefore, safety data on pregnancies exposed to this treatment are limited. CLEAR collects and describes pregnancy outcomes in this understudied population.
Efgartigimod for induction and maintenance therapy in muscle-specific kinase myasthenia gravis
The efficacy of efgartigimod in treating myasthenia gravis (MG) patients with muscle-specific kinase (MuSK) antibodies has not been demonstrated in the clinical trial, existing case reports, or observational studies.
Differentiating idiopathic Parkinson's disease from multiple system atrophy-P using brain MRI-based radiomics: a multicenter study
Differentiating idiopathic Parkinson's disease (IPD) from multiple system atrophy-parkinsonian type (MSA-P) is essential for optimizing patient care and prognosis, given the differences in disease progression and treatment response.
The use of cannabidiol as adjunctive therapy in adult patients with drug-resistant epilepsy: a systematic review and meta-analysis
Highly purified cannabidiol (CBD), recently approved for various neurological disorders, is explored as a potential therapeutic avenue for drug-resistant epilepsy (DRE) among adult people with epilepsy (PWE) in this systematic review and meta-analysis.
Fatigue, sleep quality, depression symptoms, and antiseizure medication resistance in patients with newly diagnosed epilepsy
Complaints of fatigue and poor sleep quality are common in patients with epilepsy. Fatigue may precipitate seizures, and patients with poor sleep quality have higher frequency of seizures and are more likely to have symptoms of depression.
-associated triadopathy presenting with myalgia, muscle weakness, and asymptomatic hyperCKemia
variants can alter the structure and function of the calcium channel, resulting in abnormal calcium influx and homeostasis. It is well established that pathogenic variants in can lead to hypokalemic periodic paralysis, malignant hyperthermia, and congenital myopathy. Nevertheless, the clinical presentations and disease progression of exertional myalgia and weakness associated with variants remain elusive. In this study, four affected individuals from an autosomal-dominant family were described, exhibiting symptoms of severe exertional myalgia, followed by flaccid weakness or rhabdomyolysis, along with asymptomatic hyperCKemia during the interictal period. Long exercise test showed a late decrease in compound muscle action potential amplitude. Muscle MRI revealed edema-like changes in the early stage, and fatty degeneration and substitution in prolonged disease courses, while closely aligned with the features of chronic myopathy. Ultrastructural examination revealed dilation of the sarcoplasmic reticulum and myofibrillar structural disarrangement. Genetic screening identified a c.3724A>G (p.Arg1242Gly) mutation in the gene. A literature review revealed that 15 patients exhibited the exertional myalgia and weakness phenotype associated with CACNA1S mutations, presenting similar clinical, electrophysiological, radiological, and pathological features. As the disease progressed, these patients developed severe muscle weakness, ultimately leading to wheelchair dependency. This exertional myalgia-weakness phenotype represented a unique -related phenotype that broadened the spectrum of -associated myopathy, bridging between periodic paralysis and congenital myopathies. The similarities between -associated myalgia-weakness and RyR1-associated myalgia-weakness underscored a shared pathogenesis of excitatory-contractile coupling at the triad of skeletal muscle.
Immunotherapy-mediated modulation of the gut microbiota in multiple sclerosis and associations with diet and clinical response-the effect of dimethyl fumarate therapy
Accumulating evidence supports a role of the microbiota in health and disease, including in multiple sclerosis (MS). How MS drugs affect the microbiota and whether this is part of their mode of action is yet unknown.
Evaluating natalizumab first-line and later-line use in multiple sclerosis: a US claims database analysis
Limited information exists on the healthcare resource utilization (HCRU) associated with real-world natalizumab used as a first-line (1L) versus later-line (2L+) treatment in multiple sclerosis (MS).
The potential use of plasma NfL as a diagnostic and prognostic biomarker of fatigue in early Parkinson's disease
Fatigue is a prevalent non-motor symptom that often appears in the early stages of Parkinson's disease (PD). Plasma neurofilament light chain (NfL) was elevated in PD patients and may be considered a potential biomarker for both motor and cognitive progression.
The impact of educational attainment on diagnostic and treatment delays in multiple sclerosis: a nationwide cohort study in Denmark
In multiple sclerosis (MS), the educational gradient in diagnostic and disease-modifying treatment (DMT) delays is sparsely examined, and the results are mixed.
Sequential administration of efgartigimod shortened the course of Guillain-Barré syndrome: a case series
Guillain-Barré syndrome (GBS) is a serious neurological condition with limited treatment options. A recent report demonstrated successful treatment with efgartigimod alone in two patients with GBS, although it did not significantly shorten the disease duration. This case series investigates the effects of sequential efgartigimod administration in patients with different GBS phenotypes and varying levels of disease severity. All three patients tested positive for immunoglobulin G (IgG) antibodies against serum gangliosides. In Case 1, the patient was treated with 0.4 g/kg of intravenous immunoglobulin (IVIg) for 5 days, showing minimal recovery. After receiving 3 weekly doses of efgartigimod (10 mg/kg), the patient achieved independent ambulation 19 days post-onset, with a reduction in serum ganglioside antibody titers and total IgG levels. Case 2 involved a middle-aged man with Miller Fisher syndrome (MFS)-GBS overlap, who experienced worsened autonomic dysfunction following IVIg treatment. After three doses of efgartigimod, the patient showed symptom improvement within 1 month, alongside a reduction in IgG antibody levels. In Case 3, a 27-year-old male with MFS-GBS overlap, initially unresponsive to IVIg, showed significant improvement in ophthalmoplegia following two doses of efgartigimod, with his serum ganglioside antibodies eventually becoming undetectable. Our findings suggest that sequential efgartigimod treatment may effectively reduce serum anti-ganglioside antibody titers and potentially shorten the disease course in severe GBS and MFS-GBS overlap syndrome. Additionally, it may offer clinical benefits for patients with GBS who have a poor or no response to IVIg, particularly in treating ophthalmoplegia.
Long-term efficacy and safety of alemtuzumab in participants with highly active MS: TOPAZ clinical trial and interim analysis of TREAT-MS real-world study
Alemtuzumab is a disease-modifying therapy for highly active relapsing-remitting multiple sclerosis (RRMS). Sustained efficacy up to 9 years was observed in the phase IIIb/IV open-label TOPAZ clinical trial and assessed in the real-world retrospective and prospective study, TREAT-MS.
Comparison of treatment efficacy and cost-effectiveness of rituximab and oral agents among patients with neuromyelitis optica spectrum disorders: a population-based cohort study
Rituximab (RTX) is a well-known effective treatment for neuromyelitis optica spectrum disorder (NMOSD).
Drug-induced dementia: a real-world pharmacovigilance study using the FDA Adverse Event Reporting System database
Dementia is a serious adverse event (AE) that requires attention in clinical practice. However, information on drug-induced dementia is limited. The U.S. FDA Adverse Event Reporting System (FAERS) serves as an important resource for identifying real-world adverse drug reactions and safety signals.
Crosstalk between the gut microbiota and brain network topology in poststroke aphasia patients: perspectives from neuroimaging findings
Emerging evidence indicates that gut inflammatory and immune response play a key role in the pathophysiology of stroke and may become a promising therapeutic target. However, the specific role of the microbiota-gut-brain axis in poststroke aphasia (PSA) patients remains unclear.
Oral supplementation with propionate is reflected in the serum of healthy individuals
Short-chain fatty acids (SCFAs), including propionic acid (PA), are key in immunological research. Supplementing PA has shown benefits for autoimmune diseases. A comprehensive understanding of the PA pharmacokinetics is essential for the optimal design and execution of studies utilizing orally administered PA.
Anterior nucleus of thalamus deep brain stimulation for medication refractory epilepsy modulates theta and low-frequency gamma activity: a case study
A 35-year-old gentleman with a traumatic brain injury was diagnosed with refractory epilepsy with electroencephalogram and imaging findings supporting a broad seizure onset pattern in bilateral frontotemporal regions. He therefore received a Medtronic Percept PC Deep Brain Stimulator (DBS) placed bilaterally in the anterior nucleus of the thalamus (ANT). While most refractory epilepsy patients' stimulation parameters use the SANTE trial standard clinical settings of 145 Hz, 90 μs, with cycling 1-min stimulation on and 5 min stimulation off, this participant underwent 7 different stimulation parameter tests at home following testing in the clinic of 24 different stimulation parameters across 12 neurologist visits. This device allows for simultaneous stimulation of the ANT while recording the ANT local field potential (LFP) response under different stimulation parameters. Slepian multitaper analysis, modified Fitting Oscillations, and One Over F method for detrending the aperiodic component were performed to analyze neural oscillations in the frequency domain captured in the clinic. This participant was participating in a clinical study examining the effectiveness of nonstandard DBS settings to minimize broadband neural activity in the ANT. Statistically significant neuromodulatory suppression of gamma oscillations was observed in the clinic under multiple stimulation settings. We compared the ability of these research stimulation parameters to suppress at-home ANT neural activity against the standard clinical settings and examined the effects of both sets of parameters on LFP power nonstationarity. At home, theta/alpha LFP power suppression was statistically significantly reduced under the 125 Hz, 50 μs setting as opposed to the clinical setting of 145 Hz, 90 μs. The participant has achieved greater than 50% seizure reduction for over 1 year since the last neurology visit. Suppression of gamma in the clinic in the right hemisphere and suppression of theta at home in the left hemisphere show promise as quantitative feedback biomarkers for ANT-DBS. Understanding the local and network relationships of theta and slow gamma oscillations in the thalamus would further explain how these modulated oscillations may relate to the onset and propagation of seizures.