Rose McNulty

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.

Mikkael A. Sekeres, MD, Sylvester Comprehensive Cancer Center, discusses the potential benefits of treosulfan, a newly approved agent for allogeneic hematopoietic stem cell transplantation (allo-HSCT) conditioning in acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results suggest ifetroban may improve left ventricular ejection fraction in patients with DMD.

Mikkael A. Sekeres, MD, of Sylvester Comprehensive Cancer Center, University of Miami, discussed data supporting the FDA approval of treosulfan as part of conditioning for allogeneic hematopoeitic stem cell transplantation (allo-HSCT).

Abandoning efforts to reform payment for cell and gene therapies for Medicaid beneficiaries could allow ongoing access barriers to persist, said Adam Colborn, JD, of AMCP.

Adam Colborn, JD, of AMCP, discusses the complexities of drug pricing and what policymakers must consider in efforts to reduce prices and increase access.

Adam Colborn, JD, of AMCP, discussed how President Trump's rescission of several Biden-era executive orders may impact Medicaid and Medicare initiatives.

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years in patients with Duchenne muscular dystrophy (DMD).

Prophylactic cardiac treatment may prolong survival in Duchenne muscular dystrophy (DMD), but only one-quarter of individuals received such treatment, a recent study found.

The FDA approved treosulfan in combination with fludarabine as preparation for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adult and pediatric patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

The study demonstrated a poor prognosis overall for patients with acute myeloid leukemia (AML) who were previously treated with hypomethylating agents (HMAs) for myeloid neoplasms such as myelodysplastic syndromes.

A new resource from the National Comprehensive Cancer Network (NCCN) provides evidence-based guidance on assessing and testing for inherited genetic mutations linked with increased cancer risk.

Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene abeparvovec, an investigational gene therapy.

The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with Duchenne muscular dystrophy (DMD).

Common measurements for modified adeno-associated viruses (AAVs), a gene therapy delivery method, showed substantial variation in accuracy and precision.