Health Care Delivery

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).

Katrina Ortblad, ScD, MPH, Fred Hutch Cancer Center, collaborates with researchers from the Kenya Medical Research Institute to design and test novel delivery models for HIV services.

Lynae Darbes, PhD, presented research on the effectiveness of a home-based intervention for HIV prevention among couples living in Kenya.

Cardiologists, primary care physicians, and value-based care leaders convened in Phoenix, Arizona, on November 12, 2024, to share case studies and insights on how to align cardiology with the principles of value-based care, including through the application of digital tools.

Advancing technologies have transformed the detection of heart disease and, in turn, streamlined medical processes and relieved burden on health care systems.

Molly Dean, MSW, policy advisor at Siftwell, discusses how states have encouraged managed care entities to invest in local communities, build partnerships with nonprofits, and support social determinants of health initiatives.

Xin Hu, PhD, MSPH, hypothesizes that these telemental health disparities could be driven by language barriers, historical mistrust, and systemic challenges.

A new report shows most Americans believe the government is too involved in health care regulation and want to see a ban on pharmaceutical company advertisements in the US.

Medication-assisted treatment is an excellent approach to managing addiction and related emotional symptoms, Rachel Rohaidy, MD, of Miami Neuroscience Institute with Baptist Health, explains.

This case study demonstrates how system-based tobacco cessation was enhanced in a community clinic.

The Kidney Disease: Improving Global Outcomes organization's updates to its 2024 guidelines expand SGLT2 inhibitor use and refine chronic kidney disease (CKD) management.

Despite a surge in telemental health use after the onset of the COVID-19 pandemic, minority racial/ethnic group children had lower utilization rates than White children, according to Xin Hu, PhD, MSPH.

Implementing a policy change to require preappointment surveys before scheduling initial clinic evaluations can improve wait-list times and show rates.

Elizabeth Jones, MD, FAAD, Thomas Jefferson University Hospitals, advocates for teledermatology as a vital tool to enhance health care equity by improving access for underserved populations and reducing physician burnout.