Providers

Resmetirom offers a targeted approach to metabolic dysfunction–associated steatohepatitis (MASH) for use alongside lifestyle modifications.

Charles C. Wykoff, MD, PhD, Retinal Consultants of Texas, discusses data supporting the FDA approval of revakinagene taroretcel-lwey (Encelto; Neurotech Pharmaceuticals), the first and only therapy indicated for the treatment of macular telangiectasia type 2 (MacTel).

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.

Inclusion of female and Hispanic/Latino patients has increased over time, but most trials of metabolic dysfunction-associated steatohepatitis (MASH) still take place in high-income countries and have majority-White patient populations.

Lipoprotein(a) [Lp(a)] plays a key role in assessing cardiovascular risk, making awareness efforts equally essential for prevention and early intervention.

In a recent decision, the FDA approved vutrisiran (amvuttra), making it the only approved therapy for adults with hereditary transthyretin-mediated amyloidosis (ATTR-CM) or wild-type cardiomyopathy.

Ajai Chari, MD, University of California San Francisco, explores the challenges that accompany integrating chimeric antigen receptor (CAR) T-cell therapies into multiple myeloma treatment.

Patients who did not complete a distress screener were also less likely to receive autologous stem cell transplants.

The PROMPT trials evaluate the influence of electronic health alerts for informing appropriate, evidence-based therapies to patients with cardiovascular conditions.

The rate of cGVHD-free survival was 78% at 1 year in patients who received Orca-T compared with 38% among patients who received standard allogeneic stem cell transplant for hematologic malignancies.

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.

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.

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

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.

As 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.

Advancing technologies have transformed the detection of heart disease and, in turn, streamlined medical processes and relieved burden on health care systems.

Men with melanoma tend to be diagnosed later and have worse outcomes. Artificial intelligence can help change that, a review suggests.

An oncologist from Atrium Health outlines how saving time administering immunotherapy could have far-reaching benefits.

Revakinagene taroretcel-lwey (Encelto; Neurotech), an allogeneic encapsulated cell-based gene therapy, is the first therapy to be approved for macular telangiectasia type 2.

A stratified demographics analysis of video visit telemetry data reveals that age older than 65 years and African American/Black race are associated with higher video visit failure rates, whereas language, sex, and ethnicity are not.

Nick Webb tells attendees at the Association of Cancer Care Centers that consumer experience and workforce happiness will be keys to success in the future for health systems.

Tislelizumab plus chemotherapy received FDA approval as a frontline therapy for individuals with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) whose tumors express PD-L1.

Jay T. Rubinstein, MD, PhD, University of Washington School of Medicine, discusses DB-OTO, an investigational gene therapy with potential to treat otoferlin gene–related hearing loss.

Activities of daily life, education, and employment were areas of difficulty in the transition to adulthood for patients with Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD), but those who had more siblings reported being more ready to transition to adult life.