Carol Wittlieb-Weber, MD

A forward-looking discussion on cardiac monitoring strategies, real-world data, and how vamorolone may apply across Duchenne and Becker muscular dystrophy.

In this segment, Wittlieb-Leber examines how vamorolone trials address long-standing questions about steroid exposure, cardiac risk, and meaningful cardiac endpoints in Duchenne.

In this epiosde, Carol Wittlieb-Weber, MD, provides a cardiology-focused look at why vamorolone’s dissociative steroid profile and mineralocorticoid receptor antagonism may matter for cardiac fibrosis and remodeling in DMD.

Experts discuss Duchenne muscular dystrophy's pathophysiology, disease progression, and innovative treatment options to enhance patient care and outcomes.

Experts discuss Duchenne muscular dystrophy's pathophysiology, disease progression, and innovative treatment options to enhance patient care and outcomes.

Experts discuss promising investigational therapies for muscle function improvement, including Darymiocell, which shows potential in preserving cardiac health.

Explore the complexities and advancements in gene therapy for Duchenne muscular dystrophy, highlighting treatment options and safety considerations.

Givinostat emerges as a promising oral treatment for muscle disorders, showing improved patient outcomes and broad applicability despite some side effects.

Explore the unique benefits and challenges of Vamorolone, a new steroid for Duchenne muscular dystrophy, compared to traditional treatments.

Experts discuss valuable endpoints in Duchenne clinical trials, emphasizing survival, respiratory health, and the importance of non-ambulatory patient measures.

Explore the evolving landscape of Duchenne muscular dystrophy treatments, including FDA-approved therapies and innovative alternatives for better patient care.

Explore the evolving standard of care for Duchenne Muscular Dystrophy, focusing on corticosteroids and innovative treatment strategies.

Exploring the challenges faced by families of Duchenne muscular dystrophy patients, including caregiver burdens and the importance of timely diagnosis.