ACMG Foundation Corporate Partner Insights Session - PTC Therapeutics Inc: Unlocking Potential: Innovative Advances in PKU Treatment
19 Mar 2025
Venue:
Los Angeles Convention Centre
Meeting Room:
408 B
Unlocking Potential: Innovative Advances in PKU Treatment
Please join PTC Therapeutics for an engaging session that will provide insights into the latest advancements in phenylketonuria (PKU). Case studies will highlight the unmet needs in PKU and show how an emerging therapeutic strategy may benefit different patient populations, in the context of the current treatment landscape. Lunch will be available before the session.
Topics include:
- An introduction to a novel investigational treatment for PKU, with a focus on its key differentiators from existing therapies
- Evidence to show how different patient populations may respond to different treatments
- Potential benefits of a novel investigational treatment in the dietary management of patients with PKU
Speakers
Nicola Longo, MD, PhD, Professor and Chief of Division of Clinical Genetics, Human Genetics, University of California, Los Angeles
Suzanne Hollander, MS, RD, LDN, Senior Clinical Nutrition Specialist,
Boston Children’s Hospital
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