Rose McNulty

Study participants with Duchenne muscular dystrophy (DMD) who were 2 years old at the time of treatment with delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) showed promising biomarker results and no new safety signals, according to a press release from Sarepta.1 Based […]

The phase 3 IMforte trial (NCT05091567) met its primary end points of improved independent review facility–assessed progression-free survival (IRF-PFS) and overall survival (OS) with lurbinectedin plus atezolizumab vs atezolizumab alone in patients with extensive-stage small cell lung cancer (ES-SCLC).1 Findings

Patients with lower-risk myelodysplastic syndrome (LR-MDS) treated with luspatercept (Reblozyl; Celgene Corporation) after erythroid-stimulating agent (ESA) failure in a real-world setting experienced outcomes similar to those in the MEDALIST trial (NCT02631070), according to a new study published in the British

Therapeutic innovations in cancer care have the potential to change outcomes significantly for a growing number of patients, but cost and access remain issues that must be addressed to improve care equity and overcome disparities caused by social determinants of

Cell and gene therapies (CGTs) are promising avenues of treatment for a range of diseases, but obstacles such as high costs, complex logistics, and uncertainties around reimbursement can prevent eligible patients from receiving CGTs, according to a new report from

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to slow disease progression vs a placebo in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance (QMR) measures in an exploratory analysis of the phase 3 EMBARK clinical trial (NCT05096221).1

Remote symptom monitoring (RSM) for patients with cancer was associated with a reduced risk of hospitalizations, suggesting national implementation could reduce health care utilization and complement value-based care initiatives, according to a study published in JAMA Network Open.1 In the

Patients with Duchenne muscular dystrophy (DMD) who received delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) showed long-term disease stabilization or slowed disease progression at 3 and 5 years of follow-up in data presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific

The FDA approved oral iptacopan (Fabhalta; Novartis) to reduce proteinuria in adults with C3 glomerulopathy (C3G), according to a press release from Novartis.1 Iptacopan is now the first and only FDA-approved treatment for C3G, an ultrarare, progressive kidney disease. Iptacopan

Twenty years after the initial discussions of value-based insurance design (VBID), which at its core aims to foster the use of high-value health care services while minimizing the utilization of low-value services, there is still plenty of work to be