The Framework for Embracing Gene Therapy in Hemophilia Treatment
September 30th 2023Although gene therapy is anticipated to bring positive changes for people with hemophilia, choosing to receive gene therapy will require empowerment, participation, and shared decision-making, investigators write.
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Review Highlights Potential of Neoadjuvant Immune Checkpoint Inhibition in Multiple Cancers
September 30th 2023Recent evidence suggests that neoadjuvant immunotherapy offers a unique opportunity to explore disease mechanisms and identify new biomarkers of immune checkpoint blockade response and resistance, according to the researchers.
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FDA Grants Fast Track Designation to IDE161 for BRCA+, Platinum-Resistant Advanced Ovarian Cancer
September 30th 2023The FDA has granted fast track designation to IDE161 for the treatment of adult patients with advanced or metastatic ovarian cancer harboring germline or somatic BRCA1/2 mutations who are platinum resistant and have received prior treatment with antiangiogenic and poly-ADP ribose polymerase, or PARP, inhibitor therapies.
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Altuviiio sBLA for Severe Hemophilia A in Children Accepted by FDA
September 29th 2023The supplemental biologics license application (sBLA) is supported by the results of the XTEND-Kids study, demonstrating a once-weekly 50 IU/kg dose of antihemophilic factor (recombinant) provides effective bleed protection in children with hemophilia A and can be used across clinical scenarios.
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Rapid Administration of Diazepam Results in Improved Seizure Cluster Cessation, Shorter Seizures
September 29th 2023Treatment with diazepam nasal spray resulted in a statistically significant change in time between seizure clusters, potentially reflecting a beneficial effect of intermittent rescue therapy.
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Biosimilars Could Help With Increasing Costs for AMD Therapies in Medicare
September 29th 2023Drug maker payments to ophthalmologists were associated with the choice of higher-cost therapies for age-related macular degeneration (AMD) that could increase Medicare costs, but biosimilars could help.
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Axi-Cel Yields High Responses in R/R High-Risk Large B-Cell Lymphoma
September 29th 2023Findings from the phase 2 ALYCANTE trial support axicabtagene ciloleucel as a second-line treatment for patients with relapsed/refractory (R/R) large B-cell lymphoma who are ineligible to undergo a transplant.
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Dato-Dxd Shows Survival Improvements in HR+/HER2– Breast Cancer
September 29th 2023The dual primary end points of overall survival and progression-free survival were encouraging when patients with hormone receptor–positive (HR+), HER2-negative (HER2–) breast cancer were treated with datopotamab deruxtecan.
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Subanalysis of ADAPT Reveals Consistent Efficacy With Efgartigimod Among All Genders
September 29th 2023Patients with generalized myasthenia gravis treated with efgartigimod consistently exceeded treatment compared with placebo regardless of gender in age, disease duration, body mass index, and thymectomy.
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Physical Activity Improves Performance, Frailty Scores, PROs in Multiple Myeloma
September 28th 2023Exercises such as resistance training and walking were safe and effective interventions that were associated with improved frailty scores and patient-reported outcomes (PROs) in patients with multiple myeloma receiving systemic treatment.
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Promising Results for Dupilumab in Treatment of Moderate to Severe COPD With Type 2 Inflammation
September 28th 2023New data on dupilumab demonstrate the drug’s potential usefulness in treating type 2 inflammation, a role central to respiratory diseases, including chronic obstructive pulmonary disease (COPD).
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