Latest Conference Content

Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving gene therapy.

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.

Annie Antar, MD, PhD, discusses how patients with HIV can have different and longer-lasting symptoms of COVID-19.

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care needs and physical activity barriers for individuals with myotonic dystrophy.

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

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that benefits in functional outcomes, gene expression, and muscle imaging persist 2 years after receiving the gene therapy.

Tania Gendron, PhD, speaks to the present challenges for translating biomarker discoveries to clinical practice and offers insights to how these can be overcome.

Michelle Hessen, OD, discusses how cyclosporine ophthalmic solution 0.09% is safe to use in patients with dry eye, as well as where future research should turn.

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.

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.

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.

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.

Experts share here what they take away from the Conference on Retroviruses and Opportunistic Infections (CROI) each year and why this meeting is so important in this space.

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.

Experts discussed advancements in dermatology treatment while emphasizing the value of in-person collaboration and professional rejuvenation at the American Academy of Dermatology 2025 conference.

In an interview with Brittany Craiglow, MD, FAAD, dermatologist at Dermatology Physicians of Connecticut in Fairfield, she advocates for combination therapies using baricitinib to treat pediatric alopecia areata and highlights the need for personalized treatment approaches based on Janus kinase inhibitor responses.

Lawrence F. Eichenfield, MD, FAAD, from Rady Children's Hospital and UC San Diego School of Medicine, highlighted the effectiveness of ruxolitinib cream as a nonsteroidal topical treatment for atopic dermatitis, its potential to reduce the need for systemic therapies, and the significant role of the skin microbiome in disease management.

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.

Discussions centered on mitigating the financial burden of pediatric atopic dermatitis through financial aid programs and enhancing support for young patients to independently manage their condition were emphasized at this year’s 2025 American Academy of Dermatology meeting.

Look ahead to this year's Muscular Dystrophy Association (MDA) meeting, which will feature discussions on the latest gene therapies, clinical trial data, policy considerations, and more in the realm of neuromuscular disease.

The combination of doravirine plus islatravir was noninferior to bictegravir/emtricitabine/tenofovir alafenamide and oral antiretroviral therapy in patients who switched to doravirine plus islatravir.

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.