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Ganaxolone: A Review in Epileptic Seizures Associated with Cyclin-Dependent Kinase-Like 5 Deficiency Disorder.
Paediatr Drugs
January 2025
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Oral ganaxolone (ZTALMY), a synthetic analogue of the endogenous neuroactive steroid allopregnanolone, acts as a positive allosteric modulator of synaptic and extra-synaptic γ-aminobutyric acid (GABA) type A receptor function in the CNS. In the EU and the UK, it is approved for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged 2-17 years. In a multinational phase III study (Marigold), 17 weeks' therapy with adjunctive ganaxolone, administered orally three times daily with food, significantly reduced 28-day major motor seizure frequency from baseline versus placebo in patients aged 2-19 years with CDD-associated refractory epilepsy.
View Article and Find Full Text PDFOptimizing Exclusion Criteria for Clinical Trials of Persistent Lyme Disease Using Real-World Data.
Healthcare (Basel)
December 2024
Union Square Medical Associates, 595 Buckingham Way, Suite 350, San Francisco, CA 94132, USA.
Background/objectives: Although eligibility criteria for clinical trials significantly impact study outcomes, these criteria are often established without scientific justification, leading to delayed recruitment, small sample sizes, and limited study generalizability. Persistent Lyme disease (PLD) presents unique challenges due to symptom variability, inconsistent treatment responses, and the lack of reliable biomarkers, underscoring the need for scientifically justified eligibility criteria.
Objective: This study examines the effects of commonly used enrollment criteria on sample yield in PLD clinical trials using real-world data (RWD) from the MyLymeData patient registry.
Self-Reported Delayed Adverse Events and Flare Following COVID-19 Vaccination Among Patients With Autoimmune Rheumatic Disease (AIRD) in Malaysia: Results From the COVAD-2 Study.
Int J Rheum Dis
January 2025
Department of Clinical Immunology and Rheumatology, Sanjay Gandhi Postgraduate Institute of Medical Sciences, Lucknow, India.
Objectives: To determine the prevalence of self-reported delayed adverse events (DAEs), major AEs, and flares following COVID-19 vaccinations among patients with autoimmune rheumatic diseases (AIRDs) in Malaysia.
Methodology: An electronically validated survey from the COVID-19 vaccination in autoimmune diseases (COVAD) study group was distributed in July 2021 to patients with autoimmune diseases and healthy controls (HCs). The survey collected data on DAEs (any AE that persisted or occurred after 7 days of vaccination), any early or delayed major adverse events (MAEs), and flares following COVID-19 vaccination.
An Analysis of Percutaneous Nephrolithotomy (PCNL) Performed at the Institute of Kidney Disease, Pakistan: Stone Clearance and Complications.
Cureus
December 2024
Department of Cardiovascular Medicine, Khyber Medical Institute of Medical Sciences, Kohat, PAK.
Background: Percutaneous nephrolithotomy (PCNL) is the preferred treatment for large renal stones, yet variability in outcomes arises from patient-specific factors and institutional practices. Understanding complications and predictors of success is essential to improving procedural efficacy.
Objective: This study aimed to evaluate stone clearance rates, complications classified using the Clavien-Dindo system, and predictors of PCNL outcomes, with a focus on improving lower calyx stone clearance.
Solid-phase red cell adherence versus gel column agglutination: Is there any difference for the detection of alloantibodies associated with the JK system?
Transfusion
January 2025
Blood Bank - Laboratory of Immunohematology, Centre Hospitalier Universitaire Brugmann/Université Libre de Bruxelles (ULB), Brussels, Belgium.
Background: Detection of alloantibodies associated with the JK system may be tricky. They are nevertheless associated with transfusion reactions and their detection is crucial.
Study Design And Methods: Retrospectively and over a period of 7 years, we compared the results obtained using two different assays for antibody detection.
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