Self Study - Module 1: Current Guideline Recommendations for Managing Adult Patients with Fabry Disease
May 28, 2021
The accreditation for this activity has expired.
Primary Audience:
Medical geneticists, genetic counselors, nephrologists, pediatricians, and cardiologists
Relevant Terms:
Rare disease, Fabry disease, genetics, geneticists, enzyme replacement therapy, antibodies, immunosuppression, infusion-associated reactions, lysosomal storage disorder
Ozlem Goker-Alpan, MD
Founder and President
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, VA
Ozlem Goker-Alpan, MD, is the Founder and President of the Lysosomal and Rare Disorders Research and Treatment Center (LDRTC), a nonprofit organization that focuses on lysosomal disorders and other rare diseases.
Dr Goker-Alpan founded LDRTC in 2013 with a vision of providing quality care to individuals with rare diseases by offering clinical care and clinical and translational research under one roof. LDRTC is a unique institution, and conducts a variety of clinical trials, investigator-initiated research protocols, and collaborative projects with academia, industry, and patient advocacy organizations. Another goal that LDRTC undertakes is to bring patients, expert clinicians, researchers, and industry together through education and collaboration. With that goal, LDRTC sponsored the annual Genetic, Rare & Immune Disorders Symposium (GRIDS) covering many relevant topics since 2014. Dr Goker-Alpan’s passion for translational medicine and clinical genetics research has led to LDRTC’s recognition in a short time.
Dr Goker-Alpan received her medical degree in 1990 from Marmara University School of Medicine in Istanbul, Turkey, with the highest honors as first in her class. She trained in pediatrics and then served as a Pediatric Chief Resident at The State University of New York at Stony Brook. Dr Goker-Alpan completed her first fellowship in clinical and biochemical genetics at the National Institutes of Health (NIH) Greater Washington Medical Genetics Program in 1999, and worked as an adjunct scientist at the National Child Health Institute. Her second fellowship focused on lysosomal storage disorders and Gaucher disease at the Clinical Neuroscience Branch of the National Institute of Mental Health. She coordinated the NIH Gaucher Clinic at the Medical Genetics Branch of the National Human Genome Research Institute.
As an established clinician and translational scientist in rare genetic and lysosomal storage disorders, Dr Goker-Alpan’s pursuit is to provide individualized care and treatment for patients with lysosomal storage disorders and rare diseases. Under her supervision, LDRTC has completed multiple scientific projects exploring immune pathways and lysosomal functions to develop new diagnostic and monitoring tools in lysosomal storage disorders and glucocerebrosidase-related parkinsonism. Dr Goker-Alpan serves on the scientific advisory boards of multiple pharmaceutical companies and patient advocacy organizations. Her continuing effort is to educate and train the new generation of health care providers in lysosomal storage disorders.
Anjay Rastogi, MD, PhD
Professor and Clinical Chief of Nephrology
Division of Nephrology, Department of Medicine
David Geffen School of Medicine
University of California, Los Angeles
Director, CORE Kidney Program
UCLA Health
Los Angeles, CA
Anjay Rastogi, MD, PhD, is Professor and Clinical Chief of Nephrology at the David Geffen School of Medicine at UCLA in Los Angeles, California. He completed his internal medicine residency and nephrology fellowship at UCLA under the mentorship of Nobel Laureate Professor Louis Ignarro. Dr Rastogi earned his doctorate in pharmacology from UCLA as well. He is board certified in nephrology.
Dr Rastogi is heavily involved in research. He serves as the Director of the Nephrology Clinical Research Program, and is also the Medical Director of the comprehensive UCLA Autosomal Dominant Polycystic Kidney Disease (ADPKD), Fabry Disease, Alport Syndrome, and Genetics and Kidney Disease Programs. He founded the CORE Kidney Program and the Bruin Beans Health Club at UCLA.
Dr Rastogi received the UCLA Exceptional MD of the Year Award in 2014 for his demonstration of the UCLA values of compassion, respect, excellence, discovery, integrity, and teamwork.
PROVIDERSHIP & SUPPORT
This educational activity is jointly provided by Albert Einstein College of Medicine-Montefiore Medical Center and Spire Learning.
This activity is supported by an educational grant from Sanofi Genzyme.
PROGRAM OVERVIEW
Patients with Fabry disease have a shortened life expectancy and experience a negative impact on quality of life stemming from complications of this challenging disease. With a plethora of new developments surrounding practice recommendations and approved and investigational therapies, clinicians need focused and up-to-date education on the management of this disorder. This educational activity will educate clinicians on the recent developments in Fabry disease and equip them with tools to create and implement effective, multidisciplinary, evidence-based, and patient-centered care plans to improve outcomes and quality of life.
TARGET AUDIENCE
Medical geneticists, genetic counselors, nephrologists, pediatricians, and cardiologists.
LEARNING OBJECTIVES
Upon completion of this educational activity, learners should be better able to:
- USE current guideline recommendations for patient-specific management of Fabry disease (FD) to improve cardiac and renal outcomes
- DEVELOP evidence-based, patient-specific, long-term management plans based on the efficacy, safety, and appropriateness of FD-specific and adjunctive therapeutic options
- REVIEW molecular pathways targeted by investigative therapeutics and the clinical trial data of these therapeutics
ACCREDITATION AND CREDIT DESIGNATION
In support of improving patient care, this activity has been planned and implemented by Albert Einstein College of Medicine-Montefiore Medical Center and Spire Learning. Albert Einstein College of Medicine-Montefiore Medical Center 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.
Credit Designation Statement
Albert Einstein College of Medicine-Montefiore Medical Center designates this enduring material for a maximum of 1.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
HOW TO RECEIVE CREDIT
There is no fee to participate in this activity. To receive CME credit, you must complete:
- Baseline Assessment
- Modules 1, 2, and 3
- Final Assessment
- Activity Evaluation
If you are seeking credit, you must complete the assessment and evaluation at the conclusion of the activity. Your certificate can be printed immediately.
For questions regarding this activity, please contact Albert Einstein College of Medicine-Montefiore Medical Center at cme@montefiore.org.
For technical questions related to this activity, please contact support@gathered.com.
Disclaimer
Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patients’ conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.
CONFLICT OF INTEREST STATEMENT
The “Policy on Collection of Relevant Financial Relationships and Resolution of COI” of Albert Einstein College of Medicine-Montefiore Medical Center requires that faculty participating in any CME activity disclose to the audience any relevant relationship(s) with a commercial interest.* Any presenter whose disclosed relationships prove to create a conflict of interest, with regard to their contribution to the activity, will not be permitted to present.
Albert Einstein College of Medicine-Montefiore Medical Center also requires that faculty participating in any CME activity disclose to the audience when discussing any unlabeled or investigational use of any commercial product or device not yet approved for use in the United States.
Albert Einstein College of Medicine-Montefiore Medical Center resolves all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all its educational activities. All relevant financial relationships and potential conflicts of interest have been mitigated prior to the activity.
*The ACCME defines a Commercial Interest as an entity producing, marketing, re-selling or distributing health care goods or services consumed by or used on, patients. The ACCME does not consider providers of clinical service directly to patients to be commercial interests - unless the provider of the clinical service is owned, or controlled by, an ACCME-defined commercial interest.
DISCLOSURE OF UNLABELED USE
This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.
The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.
DISCLOSURES
Faculty
Ozlem Goker-Alpan, MD
Founder and President
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, VA
Disclosure Statement:
Speaker Fees: Sanofi; Takeda Pharmaceutical Company Ltd
Grant Support: 4DMT; Amicus Therapeutics, Inc; Chiesi; Idorsia Pharmaceuticals Ltd; Protalix BioTherapeutics, Inc; Sangamo Therapeutics Inc; Sanofi
Anjay Rastogi, MD, PhD
Professor and Clinical Chief of Nephrology
Division of Nephrology, Department of Medicine
David Geffen School of Medicine
University of California, Los Angeles
Director, CORE Kidney Program
UCLA Health
Los Angeles, CA
Disclosure Statement:
Speakers Bureau: Sanofi Genzyme
Advisory Board/Consulting: Amicus; Sanofi S.A.
Research Support/Clinical Trials (paid to the institution): Idorsia Pharmaceuticals Ltd; Protalix BioTherapeutics, Inc.
Dr. Rastogi has indicated that his disclosures will not impact his ability to present an unbiased presentation.
Spire Learning
Disclosure Statement: Mairead Early and Ashton Webb, PharmD, BCACP have no relevant conflicts of interest with any ACCME-defined commercial interest.
Disclosure Statement: Jeanne Prater discloses the following:
Employee (spouse): Novo Nordisk Inc; Shareholder (spouse): Johnson & Johnson
Reviewers
Ladan Golestaneh, MD, MS (Peer Reviewer)
Professor, Department of Medicine (Nephrology)
Albert Einstein College of Medicine
Montefiore Medical Center
Bronx, NY
Disclosure Statement: Dr Golestaneh has no relevant conflicts of interest with any ACCME-defined commercial interest.
Victor B. Hatcher, PhD (CME Reviewer)
Associate Dean, Continuing Professional Development
Albert Einstein College of Medicine
Montefiore Medical Center
Bronx, NY
Disclosure Statement: Dr Hatcher has no relevant conflicts of interest with any ACCME-defined commercial interest.
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