Clinical trials are key to advancing safe and effective treatments for bleeding disorders. They are an important step in the FDA drug-approval process and provide researchers with invaluable data about a drug’s effectiveness and side effects.
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
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.
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.
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.
This introduction to gene therapy explains how genes work, and how gene therapy can be used to create new therapies for hemophilia.
FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
In this review, the authors discuss some of the primary considerations relevant to investigational gene therapies that employ adeno-associated viral
NHF's Wednesday Webinars for April 2021 will feature an array of speakers and topics of interest to both healthcare providers and patients and families with blood disorders alike.
All webinars are from 2:00pm-3:00pm ET.
In Genentech’s ongoing efforts to transparently communicate with the hemophilia community, we are sharing two updates to the Hemlibra label that were requested by the FDA.
These are exciting times in hemophilia research, with many new treatment options on the horizon. Come learn about the latest results of various phases of clinical trials on gene therapy and other new treatment options to see what the future holds for the treatment of hemophilia.
NOT AVAILABLE FOR CREDIT
Penny Kumpf, RN-BC, BSN
Von Willebrand Outreach Nurse, Hemophilia of Georgia
Kelly Tickle, MSN, APN, PPCNP-BC<
Pediatric Nurse Practitioner, Children’s Healthcare of Atlanta
Lydia Johnson, RN, BSN
Hemostasis Clinical Nurse Coordinator
SSM Cardinal Glennon Children’s Medical Center
Saint Louis Univ. Dept of Pediatrics
Kerry Hansen, RN
University of Minnesota Health, Center for Bleeding and Clotting Disorders
Jim Munn, MS, BS, BSN, RN-BC
Program Nurse Coordinator
Hemophilia and Coagulation Disorders Center, University of Michigan
By the end of this session, the participant should be better able to:
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
Stacy E. Croteau, MD, MMS
Assistant Professor of Pediatrics, Harvard Medical School Director, Hemophilia/VWD Program, Boston Children's Hospital Medical Director, Boston Hemophilia Center
Dana-Farber/Boston Children's Cancer Center & Blood Disorders Center
Christopher E. Walsh, MD, PhD
Associate Professor of Medicine, Division of Hematology and Medical Oncology
Icahn School of Medicine at Mount Sinai
Director, Hemophilia Treatment Center, Mount Sinai Hospital
New York, NY