Kyle Munz

The FDA has approved vutrisiran (Amvuttra; Alnylam Pharmaceuticals) for adult patients with cardiomyopathy classified as either hereditary transthyretin-mediated amyloidosis (ATTR-CM) or wild-type. With this announcement, vutrisiran becomes the only approved RNA interference (RNAi) intervention to mitigate cardiovascular-related hospitalizations and deaths, […]

The advent of chimeric antigen receptor (CAR) T-cell therapies has brought numerous advantages to the multiple myeloma treatment landscape; however, implementing these therapies into clinical practice comes with its own challenges. In this interview with The American Journal of Managed

The Pragmatic Trial of Messaging to Providers (PROMPT) Trials are a series of randomized studies that focus on implementing electronic medical-based alerts to enhance patients outcomes and overall care. At present, these trials are grouped into 4 divisions: lipids, heart

Addressing the cost of gene therapies has remained a prominent challenge as these interventions have become available for patients with spinal muscular atrophy, Duchenne muscular dystrophy (DMD), and other neuromuscular diseases. In this interview, Leigh Maria Ramos-Platt, MD, Children’s Hospital

Biomarker exploration has been a crucial component of amyotrophic lateral sclerosis (ALS), explained Tania Gendron, PhD, assistant professor of neuroscience, Mayo Clinic. Data at the individual level has been lacking, Gendron noted, which creates additional challenges in patient care that

The National Institutes of Health (NIH) has cancelled crucial funding for the nationwide Diabetes Prevention Program (DPP). For the last 30 years, the DPP gathered valuable data tracking patients with diabetes and prediabetes. Although the reasoning behind the move remains

Chronic inflammatory demyelinating polyneuropathy (CIDP) imposes a considerable burden on health care systems, caregivers, and patients alike. From rising financial costs to disruptions in employment and daily life, the aftermath of a CIDP diagnosis can contribute to a myriad of

At present, the FDA has approved a single gene therapy for patients with Duchenne muscular dystrophy (DMD): delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics). This therapy may be delivered through the use of an adeno-associated virus (AAV) vector. However, as trials with

Adeno-associated virus (AAV)–mediated gene therapy has particularly transformed the treatment landscape of Duchenne muscular dystrophy (DMD); however, clinical approaches with this intervention vary based on a number of factors. Here, Barry Byrne, MD, PhD, pediatric cardiologist, director, Powell Gene Therapy

The upcoming Greater Philadelphia Business Coalition on Health (GPBCH) Employer Symposia: The Heart & Other Muscles to aims to provide practical, actionable insights for managing the challenges brought about by cardiovascular and musculoskeletal (MSK) disease. This meeting, Tom Belmont, CAP,