Latest Conference Articles

One of the major highlights of the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is that invaluable networking opportunities can help clinicians elevate their own best practices.

Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating neuromuscular diseases but also raised concerns about how potential funding cuts could impact this community.

Elizabeth Jones, MD, FAAD, highlights the persistent issue of insurance companies favoring expensive, newer medications over equally effective generics in dermatology, emphasizing the time-consuming prior authorization process and advocating for patient partnerships and systemic improvements.

Rozanolixizumab is a high-affinity humanized immunoglobulin G4 monoclonal antibody and Fc receptor blocker approved to treat anti–acetylcholine receptor– and anti–muscle-specific kinase–positive generalized myasthenia gravis (gMG) in adult patients; administration is subcutaneous and takes approximately 15 minutes.

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows Duchenne muscular dystrophy (DMD) progression.

Steven Daniel Daveluy, MD, FAAD, discussed how artificial intelligence (AI) can leverage extensive patient data and guide dermatologists to improve early diagnosis and treatment of rare dermatological diseases through teledermatology.

Although the gap between low and high value has been established in many areas, insurance design has yet to adapt, according to a panel at the 20th annual Value-Based Insurance Design (VBID) Summit.

Toon Van Gorp, MD, PhD, a MIRASOL trial investigator, emphasizes that the final analysis reinforces the efficacy of mirvetuximab soravtansine-gynx (Elahere; AbbVie) in patients with folate receptor alpha-positive (FRα+), platinum-resistant ovarian cancer.

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.

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.