September 28th 2023
September 26th 2023
Although 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.
Recent 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.
The 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.
The 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.
ViiV Healthcare is developing a self-injectable HIV drug similar to Cabenuva; HHS met with insurance companies to resolve COVID-19 vaccine coverage issues; the FDA has greenlit drugmaker Novo Nordisk to add reports of ileus, or intestinal blockage, to its Ozempic label.
Patients with limited English proficiency need their mobile health (mHealth) interventions tailored more closely to their needs to improve equity of access for patients with HIV.
Treatment with diazepam nasal spray resulted in a statistically significant change in time between seizure clusters, potentially reflecting a beneficial effect of intermittent rescue therapy.
Drug 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.
Findings 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.
The 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.
Patients 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.
Exercises 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.
One meta-analysis featuring 8 studies showed that more than half of patients reported a 100% reduction in their generalized tonic-clonic seizures or tonic-clonic seizures.
Children with sickle cell disease who require hospitalization for vaso-occlusive crises can experience adverse effects for up to 12 months following hospitalization, a study found.
New 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).