Clinical

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.

Treatment guidelines in polycythemia vera currently recommend maintaining hematocrit below 45%, with a higher threshold for men vs women.

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.

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.

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.

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.

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.

Panelists discuss how social determinants of health significantly influence uncomplicated urinary tract infection (UTI) prevalence and outcomes, with patients with lower income, those from rural areas, and those lacking consistent health care access experiencing higher infection rates, delayed treatment, increased complications, and poorer overall treatment success.

Panelists discuss how their practices have observed an increasing trend in uncomplicated urinary tract infections (UTIs), particularly among younger adult women and those with recurrent infections, noting potential contributing factors such as antibiotic resistance patterns and delayed care-seeking behaviors.

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.

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

Panelists discuss how health care institutions have established structured care pathways for nonspecialist providers to evaluate mild cognitive impairment and mild dementia, incorporating validated questionnaires, standardized screening protocols, and clear referral guidelines to ensure consistent assessment across diverse clinical settings.

Panelists discuss how health care institutions employ comprehensive screening protocols for Alzheimer disease, including cognitive assessments, biomarker testing, and regular monitoring of at-risk populations, to facilitate early symptom identification and timely diagnosis that enables prompt intervention and improved patient outcomes.

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.

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 discuss which patient populations stand to benefit the most from recently approved bispecific therapies, identifying gaps or opportunities for further patient benefit, as well as the data gaps that exist in understanding the optimal use of bispecific antibodies across various cancer types.

A panelist discusses how guidelines are important for knowing when patient care starts, the progress of the patients care, and if the patient is satisfied with the response. Treatment guidelines are available for the 3 stages of hidradenitis suppurativa (HS): Hurley 1, Hurley 2, and Hurley 3. High scar scoring is also used to examine improvement in scarring over time. In real-world cases, guidelines have little to no impact on physicians treating HS, and most people often go to urgent care centers or primary care physicians when they are experiencing symptoms of HS. Health providers do not utilize guidelines except in clinical trials.

Experts discuss how the availability of bispecific therapies has influenced treatment paradigms in oncology and how bispecifics fit into current treatment algorithms for hematologic malignancies and solid tumors.

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.

These abstracts highlight the compounded challenges people with HIV face.

Flood exposure was more strongly associated with increased health care use and cost during the summer months and among Medicare beneficiaries aged 85 or older.

The authors argue that the stimulant shortage is being perpetuated by the overdiagnosis of attention-deficit/hyperactivity disorder (ADHD) in adults because executive functioning deficits are not included in diagnostic criteria.