Rose McNulty

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.

Debra Patt, MD, PhD, MBA, executive vice president of Public Policy and Strategy for Texas Oncology, said the practice received positive feedback from nurses and patients during a pilot that concluded in February.

The new findings are the first to describe distributions and perturbations of low-density granulocyte and normal-density neutrophil subsets in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and idiopathic cytopenia of unknown significance.

The study found that just 20% of trials submitted to both the FDA and European Medicines Agency had matching evidence.

Revakinagene taroretcel-lwey (Encelto; Neurotech), an allogeneic encapsulated cell-based gene therapy, is the first therapy to be approved for macular telangiectasia type 2.

Enhanced premium tax credits (PTCs) have made marketplace health insurance more affordable, and eliminating them could have sweeping impacts on consumers and the health care industry, according to a new report.

Tislelizumab plus chemotherapy received FDA approval as a frontline therapy for individuals with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1.

Jay T. Rubinstein, MD, PhD, University of Washington School of Medicine, discusses DB-OTO, an investigational gene therapy with potential to treat otoferlin gene–related hearing loss.

Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD), but those who had more siblings reported being more ready to transition to adult life.

Ten children who received DB-OTO for profound genetic hearing loss due to variants of the otoferlin gene showed notable improvements in hearing, according to initial results of the CHORD trial.

At 2 years, participants in the compensated MASH cirrhosis treatment arm of the MAESTRO-NAFLD-1 trial experienced a statistically significant 6.7 kPa reduction in liver stiffness from baseline.

In the phase 1/2 INSPIRE DUCHENNE trial, interim data showed an average microdystrophin expression of 110% among participants with Duchenne muscular dystrophy (DMD) at 90 days post treatment.

The next-generation sequencing (NGS)–based AlloHeme test accurately predicted relapse following allogeneic hematopoietic stem cell transplantation (allo-HSCT) in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS).

Anti-acetylcholine receptor antibody (AChR-Ab) titers and the AChR-Ab rates of change correlated with myasthenia gravis disease severity scores in a recent study.

The FDA approved Amneal Pharmaceuticals' generic lenalidomide capsules, referencing Celgene's Revlimid, in multiple strengths.

Patient-reported outcomes (PROs) were similar among those treated with tivozanib monotherapy and those given combination tivozanib and nivolumab in for renal cell carcinoma (RCC) in the TiNivo-2 study, according to a poster presented at the American Society of Clinical Oncology Genitourinary Cancers Symposium by Katy Beckermann, MD, PhD.

Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of osteoporosis in adult men with Duchenne muscular dystrophy (DMD).

Cell and gene therapies can be life-changing for patients with certain conditions, but the process of receiving them poses barriers for patients and caregivers that require multistakeholder solutions, according to a white paper from the National Pharmaceutical Council.

Patients with progressive pulmonary fibrosis who received nerandomilast showed improved forced vital capacity from baseline at 52 weeks in the phase 3 FIBRONEER-ILD trial.