FDA places the Pfizer/Sangamo Therapeutics phase 3 AFFINE haemophilia A gene therapy study on clinical hold
This activity is intended for hematologists, pediatricians, primary care physicians, nurses, and nurse practitioners. It is part of Medscape’s series “Clinical Advances in Gene Therapy for Hemophilia.” Presented through a collaboration between Medscape and the National Hemophilia Foundation, the series includes various discussions relevant to the latest data on gene therapy as well as the practicalities associated with using gene therapy for hemophilia.
Whether you or someone in your family has been recently diagnosed with hemophilia, or you've lived with hemophilia your entire life and want a refresher on the fundamentals, this session is for you. We will explain what hemophilia is, how it's treated, and what issues may arise at different life stages.
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.
The U.S. Food and Drug Administration (FDA) has partially rescinded a pair of supplemental Biologics License Applications (BLAs) the agency had granted, in error, for two recombinant factor IX products, BeneFIX® (Pfizer) and IXINITY (Aptevo).
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.
Hemophilia is a uniquely challenging and sometimes perplexing condition for physical therapists to manage and treat. Physical therapists who see patients across the lifespan may manage conditions that range from athletic injuries to frailty. Clinicians often rely on reviewing limited evidence, consulting colleagues, and ultimately critical thinking and best clinical judgment to develop a management plan. This session will outline a framework for different scenarios seen in the clinic and look at six broad management schemes.
Let’s face it, adolescence is a challenging time even under the best of circumstances. As parents of teens with hemophilia and an inhibitor, one of those biggest challenges, preparing to let go, can be especially difficult. As children grow into their teen years, parents undergo a transition of their own, from advocates and fierce protectors to ones willing, in fits and starts, to take a step back. In this webinar, a social worker will join a teenager, young adult and mom to discuss this evolution and techniques for engendering independence.
What exactly is meant by individualized therapy? What might be the future treatment and quality-of-life implications for hemophilia patients? Learn more about this state-of-the-art approach to care, and how it could improve the health of a person living with hemophilia.
Transcription of "One Size Does Not Fit All: Individualized Therapy"
Whether within friendships, romantic relationships, at school or in the workplace, an adult with hemophilia will confront the question of disclosing their bleeding disorder status many times over. Join us to learn techniques that will help you negotiate this process in the age of digital and social media.
Transcription of "Is Hemophilia a Secret? Disclosure in a World Filled with Technology"
It is a reality familiar to most adults with hemophilia of a certain age: repeated joint bleeds which subsequently causes joint deterioration, affecting everyday quality of life. But the story does not have to end there. Learn how prophylactic therapy can be used to halt the momentum of degenerative joint damage, decrease pain and help you regain mobility.
Transcription of "The Advantage of Prophylaxis for Adults with Joint Issues"
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
This activity is intended for emergency medicine physicians, hematologists, pediatricians, nurses, and other healthcare professionals who manage patients with hemophilia. The goal is to increase understanding of the optimal management of these patients when they present to the emergency department (ED). It was developed through a partnership between Medscape Education, the American College of Emergency Physicians, and the National Hemophilia Foundation.
Upon completion of this activity, participants will:
This activity features an international panel of experts that review the science underlying therapy strategies, provide updates on the latest data for therapies in development, and critically evaluate the potential role gene therapy may play in patient care, both now and in the future. It is intended for hematologists, pediatric hematologists, and all members of the clinical care team involved in the care of patients with hemophilia.