Rose McNulty

Delandistrogene moxeparvovec demonstrated a manageable safety profile across clinical trials for Duchenne muscular dystrophy (DMD), with most adverse events emerging within 90 days of infusion.

Pieter Sonneveld, MD, PhD, professor of hematology and chair of the Erasmus MC Cancer Institute in Rotterdam, Netherlands, discussed the continued use of subcutaneous daratumumab following initial treatment, as well as the changing landscape of multiple myeloma treatment.

Despite holding promise in a range of disease states, a variety of challenges prevent cell and gene therapies (CGTs) from more widespread use, according to a new report from Cardinal Health.

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients with Duchenne muscular dystrophy (DMD) based on muscle quantitative magnetic resonance measures.

Remote symptom monitoring in cancer care reduces hospitalizations, enhances value-based care, and supports diverse patient populations, according to new findings.

Pieter Sonneveld, MD, PhD, chair of the Erasmus MC Cancer Institute, discussed the findings of a study modeling long-term progression-free survival (PFS) in patients with multiple myeloma (MM) treated with a daratumumab quadruplet regimen.

Infection was a leading cause of death in lower-risk myelodysplastic syndromes (MDS), highlighting the need for vigilant monitoring and preventive strategies.

Prademagene zamikeracel (Zevaskyn) is the first and only cell-based gene therapy for wound treatment in patients with recessive dystrophic epidermolysis bullosa.

Adding venetoclax to decitabine-cedazuridine significantly enhanced response rates in patients with higher-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia.

Researchers are calling for more targeted efforts to improve health equity after a new analysis revealed that cancer symptom documentation and burden vary across certain demographics.

Sleep duration and sleep pattern score were both independently associated with obesity occurrence, and there was a nonlinear relationship between obesity and sleep duration.

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

The remote measurement tool enables Duchenne muscular dystrophy (DMD) assessments through analysis of caregiver-recorded videos.

Elinzanetant significantly reduced the frequency and severity of vasomotor symptoms, known as hot flashes, compared with placebo across body mass index (BMI) and smoking status subgroups.

While findings around hospital and emergency department use were similar at the individual and neighborhood levels, the use of outpatient services differed.

Coverage from the San Francisco Regional session of the Institute for Value-Based Medicine.

Orca-T showed lower rates of graft-vs-host disease or infection compared with allogeneic hematopoietic stem cell transplantation for myelodysplastic syndrome (MDS) or acute leukemias in the Precision-T trial, Caspian Oliai, MD, MS, UCLA Bone Marrow Transplantation Stem Cell Processing Center, said.

None of the eteplirsen-treated patients reached a left ventricular ejection fraction below 50% compared with 22.1% of patients in the control group.

Study participants treated with elinzanetant experienced significantly reduced hot flash frequency vs paroxetine, desvenlafaxine, and gabapentin in a new meta-analysis.

The findings indicate that the currently approved 600-mg dose of intravenous ocrelizumab is optimal to significantly slow disability progression for patients with multiple sclerosis (MS).

Carla Nester, MD, MSA, FASN, is coinvestigator for the ongoing APPEAR-C3G trial (NCT04817618), data from which were used to support the FDA’s recent approval of iptacopan (Fabhalta; Novartis) in complement 3 glomerulopathy (C3G).

Hospitalized patients with opioid use disorder (OUD) who received in-hospital addiction consultation services were more likely to receive evidence-based OUD care, a new study found.

Giulio Cossu, MD, University of Manchester, discusses barriers to gene therapy research and development.

Inebilizumab-cdon (Uplizna; Amgen) was approved as the first and only treatment for immunoglobulin G4–related disease (IgG4-RD), a chronic and debilitating immune-mediated inflammatory condition.

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for patients with Duchenne muscular dystrophy (DMD).

Erythropoiesis-stimulating agent (ESA) treatment before or early after regular transfusion therapy improved overall survival (OS) In lower-risk myelodysplastic syndromes (MDS), a new study found.

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients with Duchenne muscular dystrophy (DMD) in the phase 2 FORWARD-53 trial.

Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, spoke to the current state of research on revakinagene taroretcel in macular telangiectasia type 2 (MacTel), as well as the importance of patient groups for the rare condition.