Rose McNulty

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.

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found tolerable and showed signs of efficacy in a real-world cohort.

The approval of revakinagene taroretcel (Encelto; Neurotech) addresses a significant unmet need for patients with macular telangiectasia type 2 (MacTel), clinical investigator Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, said.

Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, discusses data supporting the FDA approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals), the first and only therapy indicated for the treatment of macular telangiectasia type 2 (MacTel).

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows Duchenne muscular dystrophy (DMD) progression.

Iptacopan's (Fabhalta; Novartis) approval makes it the first and only treatment approved for C3 glomerulopathy.

Although the gap between low and high value has been established in many areas, insurance design has yet to adapt, according to a panel at the 20th annual Value-Based Insurance Design (VBID) Summit.

The rate of cGVHD-free survival was 78% at 1 year in patients who received Orca-T compared with 38% among patients who received standard allogeneic stem cell transplant for hematologic malignancies.

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.