April 23rd 2025
Emergency departments (ED) struggle to screen for social determinants of health, inhibiting quality care and impacting health disparities among vulnerable populations.
Neurologists Share Tips for Securing Patient Access to Gene Therapies
March 19th 2025Tenacious 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.
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Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
March 18th 2025The 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.
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Shaping Gene Therapy Approaches in Neuromuscular Disease
March 18th 2025Adeno-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.
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How Mirdametinib Improves QOL for People With NF1-Associated Plexiform Neurofibromas
March 18th 2025Christopher 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.
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Solving the Transition Conundrum as More Children With Muscular Dystrophy Live to Adulthood
March 17th 2025Learning 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.
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For Neuromuscular Disease Community, an Era of Opportunities and Threats
March 17th 2025Robert 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.
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Oz Confirmation Hearing Probes Vision for Medicaid but Coalesces Around Well-Being
March 14th 2025Mehmet 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.
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PAH Treatment Benefits Extend to Patients With Repaired Congenital Heart Disease
March 14th 2025Historical 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.
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Study Limitations, Future Research Directions on Telemental Health Disparities Among Children
March 14th 2025Areas 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.
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As Claims Denials Surge, AI and Data-Driven Insights Equip Clinicians, Hospitals to Fight Back
March 13th 2025As 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.
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Regenerating Neurons, Muscle, and Hope in the Field of Muscular Dystrophy
March 13th 2025The 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.
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Closing the Gaps in Cardiac Care With Technology, Collaboration
March 12th 2025Cardiologists, 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.
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States Call for Managed Care to Prioritize Rural Health
March 12th 2025Molly 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.
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Improving System-Based Tobacco Cessation in a Community Health Clinic
This case study demonstrates how system-based tobacco cessation was enhanced in a community clinic.
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