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Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic advances in treating spinal muscular atrophy (SMA) are not uniformly making it into the hands of patients who could benefit.

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

Annie Antar, MD, PhD, discusses the association between the symptoms of long COVID and HIV status.

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.

Elizabeth Jones, MD, FAAD, Thomas Jefferson University Hospitals, highlights the continued relevance of older, generic dermatologic therapies despite the availability of newer, targeted treatments.

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.

The Conference on Retroviruses and Opportunistic Infections 2025 (CROI) featured multiple sessions that focused on breaking research and treatments.

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

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.

The final analysis showed similar overall survival, progression-free survival, and objective response rates, with no new safety signals identified compared with the primary analysis.

A new analysis out of England shows a considerable level of uncertainty among adults who think they could have long COVID, as well that levels of the chronic condition are disproportionately higher among certain socially disadvantaged groups.

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.

The final data analysis confirmed that mirvetuximab soravtansine-gynx (Elahere; AbbVie) significantly improves progression-free survival, overall survival, and objective response in patients with folate receptor alpha-positive (FRα+) platinum-resistant ovarian cancer.

Cemacabtagene ansegedleucel, an allogeneic chimeric antigen receptor (CAR) T-cell therapy, is being investigated in relapsed/refractory large B-cell lymphoma.

Data on care satisfaction, ease managing care, and out-of-pocket spending were the outcomes of interest for this new analysis that investigated the relationship between healthcare utilization and beneficiary experience within traditional Medicare.

As St. Patrick's Day brings global celebrations involving alcohol, Ireland looks forward to rethinking excess consumption with a new tool set to arrive next year: the world's most comprehensive alcohol warning label.

Tom Belmont, president and CEO, Greater Philadelphia Business Coalition on Health (GPBCH) previews the upcoming employer symposia, which seeks to help employers navigate solutions for the management and prevention of cardiovascular and musculoskeletal disease.

The FDA first approved eculizumab for use in adult patients with generalized myasthenia gravis in 2017, before expanding the indication to include pediatric patients who are 6 years or older and positive for antiacetylcholine receptor antibodies.

This new study has found perceived discrimination in health care leads to delayed visits, especially among younger and minority patients.

These studies published in The American Journal of Managed Care® explored telehealth, care access, and patient experience during the COVID-19 pandemic.

Michael Chernew, PhD, professor of health care policy and director of the Healthcare Markets and Regulation Lab, Harvard Medical School, shares how cost-sharing policies shape access to critical health care services and influence value-based insurance design.

Weight loss drugs like tirzepatide and semaglutide are growing in popularity and offer significant health benefits, but they are not cost-effective at their current prices, according to a new evaluation, placing economic burdens on patients with obesity in the US.

Patients expressed concern that overreliance on artificial intelligence (AI) could lead to clinicians losing their skills, both medical and interpersonal.

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.

National MS Education and Awareness Month is celebrated each year in the US in March, after having been launched in 2003 by MS Focus: the Multiple Sclerosis Foundation and affiliated groups.

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing spinal muscular atrophy (SMA).

The role of artificial intelligence, DataDerm, and telehealth in advancing dermatology care was discussed throughout the meeting, with experts highlighting their potential regarding patient access and health equity.

The new findings are the first to describe distributions and perturbations of low-density granulocyte and normal-density neutrophil subsets in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and idiopathic cytopenia of unknown significance.