Recent Advances in Gene Therapy for Hemophilia
Target Audience: Pharmacist
Course Overview
Hemophilia is a hemorrhagic disease characterized by a deficiency in blood clotting. It results from gene mutations in the genes that encode clotting factor proteins that form blood clots. The disease manifests itself in excessive and prolonged periods of external and internal bleeding. The conventional hemophilia treatment replaces missing blood clotting factors through intravenous infusion of plasma and cryoprecipitates (plasma-derived) and recombinant clotting factor concentrates. While these agents and treatments are effective, the current gene therapy strategies may offer new alternatives to the replacement protocols. Recent gene therapy directions, including viral, non-viral, and gene-editing technologies, are often more effective and provide new therapeutic avenues for routine hemophilia treatment.
Learning Objectives
Upon completion of this educational activity, participants should be able to:
- Provide an overview of hemophilia types and characteristics
- Describe the diagnosis of hemophilia
- Describe the current treatment options for hemophilia
- Describe pharmacological gene therapy approaches for hemophilia
- Discuss current challenges in gene therapy for hemophilia
Faculty
I. Introduction
II. Definition and Prevalence of Hemophilia
III. Causes of Hemophilia
IV. Symptoms of Hemophilia
V. Diagnosis of Hemophilia
VI. Other Treatment Products
VII. Gene Therapy of Hemophilia
VIII. Challenges of Gene Therapy
IX. Patient Counseling
X. Future Trends in Gene Therapy
XI. Summary
From September 12, 2023 through September 12, 2026, participants must:
- Read the "learning objectives" and "author and planning team disclosures"
- Study the section entitled "educational activity"
- Complete the Course Test and Evaluation form. The Course Test will be graded automatically. Following successful completion of the Course Test with a score of 70% or higher, a statement of participation will be made available immediately. (No partial credit will be given.)
Credit for this course will be automatically uploaded to CPE Monitor®.
Faculty Planner Disclosure
The following individuals were involved in developing this activity: Salam Kadhim, PhD, and Pamela Sardo, PharmD, BS. Salam Kadhim, PhD, was a senior scientist at Inmed Pharmaceuticals until July 1, 2022. Pamela Sardo, PharmD, BS, was an employee of Rhythm Pharmaceuticals until March 2022. There are no financial relationships relevant to this activity to report or disclose by any of the individuals involved in the development of this activity.
Unlabeled Use Disclosure
The information provided in this course is general in nature and it is solely designed to provide participants with continuing education credit(s). This course and materials are not meant to substitute for the independent, professional judgment of any participant regarding that participant’s professional practice, including but not limited to patient assessment, diagnosis, treatment and/or health management. Medical and pharmacy practices, rules, and laws vary from state to state, and this course does not cover the laws of each state; therefore, participants must consult the laws of their state as they relate to their professional practice. Healthcare professionals, including pharmacists and pharmacy technicians, must consult with their employer, healthcare facility, hospital, or other organization, for guidelines, protocols, and procedures they are to follow. The information provided in this course does not replace those guidelines, protocols, and procedures but is for academic purposes only, and this course’s limited purpose is for the completion of continuing education credits. Participants are advised and acknowledge that information related to medications, their administration, dosing, contraindications, adverse reactions, interactions, warnings, precautions, or accepted uses are constantly changing, and any person taking this course understands that such person must make an independent review of medication information prior to any patient assessment, diagnosis, treatment and/or health management. Any discussion of off-label use of any medication, device, or procedure is informational only and such uses are not endorsed hereby. Nothing contained in this course represents the opinions, views, judgments, or conclusions of RxCe.com LLC. RxCe.com LLC is not liable or responsible to any person for any inaccuracy, error, or omission with respect to this course, or course material.
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Accreditation
In support of improving patient care, RxCE.com is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.