Marla Weetall
Senior Vice President, Pharmacology and Biomarkers PTC Therapeutics
Marla Weetall is Senior Vice President, Pharmacology & Biomarkers at PTC Therapeutics, where she has led pharmacology and biomarker strategy for more than 20 years, supporting the development of therapies for rare genetic diseases. She has played a key role in advancing PTC’s Duchenne muscular dystrophy (DMD) portfolio, including Translarna® (ataluren), the first therapy developed for nonsense mutation DMD, and Emflaza® (deflazacort), a corticosteroid approved for the treatment of DMD. With expertise spanning translational science, preclinical pharmacology, and biomarker development, Marla helps bridge laboratory discoveries with clinical outcomes. Prior to joining PTC, she held research positions at Novartis and Hoffmann-La Roche and earned a Ph.D. in Chemistry and a B.S. in Biology from Cornell University.
Seminars
As the landscape of therapeutic approaches for muscular disorders expands, so must the endpoints used to assess them. Demonstrating meaningful clinical benefit remains one of the biggest barriers to successful approval, and while functional assessments continue to play an important role, sponsors are exploring novel biomarkers, digital measures and disease-specific endpoints that can provide earlier, more sensitive evidence of treatment effect. Attendees will gain practical insights into endpoint selection, validation and implementation from experts navigating today’s evolving regulatory landscape.
This workshop will:
- Explore the shortfalls of traditional functional assessments such as the 6-minute walk test and the North Star Ambulatory Assessment to identify the key challenges like variability in a largely heterogeneous patient population
- Discuss aligning endpoint selection with disease biology to demonstrate clinically meaningful benefits
- Discuss novel strategies including digital endpoints to detect treatment effects earlier and generate evidence that supports regulatory decision-making
