Health Care Delivery

This commentary explains why comparing a launch price with a value-based price from a cost-effectiveness analysis requires further examination.

In a recent decision, the FDA approved vutrisiran (amvuttra), making it the only approved therapy for adults with hereditary transthyretin-mediated amyloidosis (ATTR-CM) or wild-type cardiomyopathy.

Ajai Chari, MD, University of California San Francisco, explores the challenges that accompany integrating chimeric antigen receptor (CAR) T-cell therapies into multiple myeloma treatment.

Addressing patients with chronic kidney disease requires a commitment to data, education, and community, specifically in those affected by social determinants of health (SDOH).

Patients who did not complete a distress screener were also less likely to receive autologous stem cell transplants.

The PROMPT trials evaluate the influence of electronic health alerts for informing appropriate, evidence-based therapies to patients with cardiovascular conditions.

A study of 200 patients with chronic lymphocytic leukemia (CLL) showed a lower rate of severe and serious adverse events among those treated with zanubrutinib compared with ibrutinib.

To better understand patient experiences, the researchers analyzed qualitative and quantitative data from their social media posts about uncomplicated urinary tract infections (UTIs).

Tenacious efforts at every level, from the individual clinician to the hospital to the state to Congress, will be needed to make sure patients can access life-saving gene therapies for neuromuscular diseases.

Medication costs are only a piece of the puzzle when clinicians think about health care access, explains Leigh Maria Ramos-Platt, MD.

Recent cuts to federal grants are set to have a widespread impact across numerous scientific and medical studies.

Chronic inflammatory demyelinating polyneuropathy (CIDP) can have a substantial impact on patients everyday life and, consequently, put added stress on health care systems.

The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but also logistical hurdles and a need for complex discussions between clinicians and families.

Adeno-associated virus (AAV)–mediated gene therapy was one of the focal points of this year's Muscular Dystrophy Association conference. Here, Barry Byrne, MD, PhD, University of Florida, speaks to the novel development of AAV gene therapy and its mechanism of action.

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

Christopher L. Moertel, MD, University of Minnesota, discusses the trial data that helped get mirdametinib approved and what providers can do to keep measuring quality of life (QOL) improvements.

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach adulthood thanks to transformative therapy advances.

Robert Califf, MD, former commissioner of the FDA, delivered a keynote address at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference that highlighted the enormous opportunities for progress in neuromuscular disease care amid a changing policy environment.

Mehmet Oz, MD, the nominee to lead CMS under the Trump administration, testified in a confirmation hearing before the Senate Finance Committee, where he found common ground on improving outcomes through healthier lifestyle choices but encountered repeated questions on potential Medicaid cuts.

Historical data show the prevalence of pulmonary arterial hypertension (PAH) in adult patients with congenital heart disease ranges from 4% to 28%, according to studies from the US and Europe—and that the prevalence of these comorbid condition is on the rise.

Areas for further research suggested by Xin Hu, PhD, MSPH, include examining differences in telemental health uptake between new and existing patients and exploring how these differences vary across demographic subgroups.

As health care costs continue to rise and the burden of chronic disease grows, data-driven insights will be essential in shaping the future of patient care, according to experts from Komodo Health and SmarterDx.

The 2025 Muscular Dystrophy Association Clinical & Scientific Conference, convening in Dallas, Texas, from March 16-18, will feature clinical updates, expert insights, and breaking trial findings that sum up to a new frontier of care for neuromuscular diseases.

Priscilla Tsondai, MD, MPH, breaks down top care gaps as opportunities for care improvement.

The invasive nature of lipid biopsies has lead researchers to dedicate more efforts to unveiling noninvasive diagnostic methods for metabolic dysfunction–associated steatohepatitis (MASH).