Rose McNulty

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.

The approval marks the second for a generic in this class of medications indicated to improve glycemic control in patients with type 2 diabetes as a complement to diet and exercise.

In the final part of our interview, Ticiana Leal, MD, Winship Cancer Institute, looks to the future of perioperative care in the non–small cell lung cancer (NSCLC) arena and the importance of risk stratifying patients.

Ticiana Leal, MD, Winship Cancer Institutes, explains the importance of biomarker testing and multidisciplinary conversations while caring for patients with early-stage non–small cell lung cancer (NSCLC).

While previous trials have led to questions surrounding the potential benefits of valve interventions in mitral or tricuspid regurgitation, a pair of trials presented recently demonstrate their potential, Javed Butler, MD, MPH, MBA, president of the Baylor Scott and White Research Institute, said.

HDP-101, a novel antibody-drug conjugate, targets BCMA with a synthetic amanitin payload that causes tumor cell death.

In the phase 2 FORWARD-53 study, the exon-skipping oligonucleotide WVE-N531 showed promising safety and efficacy in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping.

Daratumumab plus VRd and frontline daratumumab plus VTd produced deep, durable responses in patients with transplant-eligible multiple myeloma in updates to the PERSEUS and CASSIOPEIA trials, respectively.

JT Lew, PharmD, MBA, managed care pharmacist at MultiCare Health System, spoke to the impacts of processes such as prior authorization and step therapy requirements in the realm of multiple sclerosis (MS).

Bimekizumab-bkzx (Bimzelx) is now FDA approved for 4 chronic immune-mediated inflammatory diseases: psoriatic arthritis, nonradiographic axial spondyloarthritis (axSpA), ankylosing spondylitis, and moderate to severe plaque psoriasis.

New findings add to the understanding of the patient experience in axial spondyloarthritis (axSpA), which can help inform patient-centered clinical care.

Cases of atrial arrhythmia in patients with Duchenne muscular dystrophy (DMD) are common but lack a standard solution, and more long-term data on the management of arrhythmias in DMD are needed.