Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect?

Introduction: Duchenne muscular dystrophy (DMD) is a severe X-linked disorder characterized by progressive muscle weakness and eventual death due to cardiomyopathy or respiratory complications. Currently, there is no cure for DMD, with standard treatments primarily focusing on symptom management. Using immunosuppressive measures and optimized vector designs allows for gene therapies to better address the genetic cause of the disease.

Frequency of ischemic cardiac events in patients receiving long-term multikinase inhibitor: A report of three cases.

Objective: To investigate the incidence and characteristics of ischemic cardiac events, specifically major adverse cardiac events (MACE), in patients undergoing long-term treatment with multikinase inhibitors (MKIs) such as lenvatinib and sorafenib.

Methods: A single-center retrospective analysis was conducted on 41 patients treated with lenvatinib or sorafenib for more than one year at our institution from 2015 to 2022. Patient records were reviewed to collect data on demographics, cancer type, cardiovascular risk factors, MKI treatment duration, and MACE incidence.

Associations of sarcopenia and malnutrition with 30-day in-hospital morbidity and mortality after cardiac surgery.

Objectives: Sarcopenia and malnutrition often occur simultaneously in adults with cardiovascular diseases. Our objective was to determine the associations of preoperative sarcopenia and malnutrition with major adverse cardiac and cerebral events (MACCE) after cardiac surgery.

Methods: We retrospectively analysed 154 consecutive patients who underwent elective cardiac surgery between January 2015 and June 2018 at two institutions in Japan.

An Updated Analysis of Exon-Skipping Applicability for Duchenne Muscular Dystrophy Using the UMD-DMD Database.

Background/objectives: Antisense oligonucleotide (ASO)-mediated exon-skipping is an effective approach to restore the disrupted reading frame of the dystrophin gene for the treatment of Duchenne muscular dystrophy (DMD). Currently, four FDA-approved ASOs can target three different exons, but these therapies are mutation-specific and only benefit a subset of patients. Understanding the broad applicability of exon-skipping approaches is essential for prioritizing the development of additional therapies with the greatest potential impact on the DMD population.