The Basics of Innovative Therapies: What You Need to Know

We continue to see significant research and innovation in the treatment of Hemophilia.  It is becoming more important than ever to stay informed on all the advancements and innovation  – what they are, how they work in the body and how safe they are.   Understanding all the options including currently approved treatments, the pros and cons and the promise and limitations of each type of therapy will enable community members to have informative conversations with their providers and to ultimately make the best decision on treatment.  This presentation is designed to provide a basic overview

Gene Therapy Basics for Hemophilia: What You Need to Know

Recently there has been an explosion of innovative research in the treatment of Hemophilia utilizing gene therapy technology. This workshop will help you develop a basic understanding of the complexities of gene therapy including terminology, how gene therapy works, and identifying the potential, limitations, risks and unknowns associated with gene therapy. We also provide a brief introduction to other novel therapies and an overview of what treatments are currently approved.

Gene Therapy: A Candid Conversation

The realization that gene therapy could be just around the corner is both daunting and exciting. Join us for a balanced, patient-centric discussion on gene therapy from multiple perspectives: a provider, a patient in a gene therapy clinical trial, a patient on a newer therapy, and a patient who is satisfied with conventional factor replacement therapy.

Gene Therapy: Getting Up to Speed

Advancements in gene therapy are happening at a fast clip. Having a solid understanding of this technology is important to patients, caregivers and families. Join us for an in-depth discussion of gene therapy including historical aspects, key terms, various approaches to the technology, and how it may revolutionize hemophilia treatment.

Forging a New Path – Gene Therapy Readiness and HTC Implementation



Steven W. Pipe, MD, Faculty Chair
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases,
Pediatric Medical Director, Hemophilia and Coagulation Disorders Program,
Director, Special Coagulation Laboratory,
University of Michigan
Ann Arbor, Michigan