Novel treatment strategies for upper-tract urothelial cancer

Wednesday, March 23, 2022; 8:00-9:30AM CDT

Meeting room Davidson A
Music City Center
201 Rep. John Lewis Way S., Nashville, TN 37203
Nashville, TN, USA

Live streamed at  ACMG 2022 Congress


Registration is obligatory for access to this satellite symposium, and is part of the general congress registration; please register here for both the satellite symposium and the congress.


At the conclusion of this session, participants should be able to:

  • Recognize the importance of inclusion of females in clinical trials of FD and of addressing their specific needs in the trial design
  • Describe the symptoms at onset of FD in heterozygous female patients
  • Explain how early initiation of FD-specific treatment benefits heterozygous females
  • Utilize methods for optimizing treatment strategies for females with FD, including referral, early initiation, and follow-up


A CME-accredited on-demand webcast of this satellite symposium will be available for one year on Ology Medical Education.



Dear Colleague,

Welcome to our satellite symposium entitled A multidisciplinary conversation on the stories of women with Fabry disease, organized in conjunction with the ACMG Annual Clinical Genetics Meeting 2022.

Diagnosis of Fabry disease is frequently delayed in female patients who often present with variable symptoms and who were historically considered unaffected carriers of Fabry disease. This complicated clinical picture and late diagnosis frequently results in delayed implementation of disease management and treatment. During this satellite symposium our expert faculty will illuminate key aspects of comprehensive care and treatment of female patients with Fabry disease, from diagnosis to development of optimal treatment plans and future clinical trial design.

This hybrid event features ample opportunity for interaction between participants and faculty, with time to ask questions and televoting on key issues. The patient voice, which is so vital in the development of patient-centric care, can be heard in patient videos shown at different moments during the symposium to guide the flow of the expert discussion.

We hope that you will find this innovative symposium both informative and thought-provoking, and that you will gain new insights that you can apply in your daily clinical practice when managing your female patients with Fabry disease.

With warm regards,

Dawn A. Laney, MS, CGC, CCRC
Emory University School of Medicine
Emory University
Atlanta, GA, USA



Contact information for questions about the activity:,,

Wednesday, March 23, 2022
Meeting room: Davidson A

8:00-8:10 CDT Welcome and introduction
Dawn A Laney (Atlanta, GA, USA)
8:10-8:35 CDT Illuminating the Path to Diagnosis of FD in Females
Dawn A Laney (Atlanta, GA, USA)
8:35-9:05 CDT Developing Optimal Treatment Plans for Females with FD
Anjay Rastogi (Los Angeles, CA, USA)
9:05-9:25 CDT Designing the Clinical Trials of Tomorrow
Robert J Hopkin (Cincinnati, OH, USA)
9:25-9:30 CDT Key Messages and Close of Meeting
Dawn A Laney (Atlanta, GA, USA)

Dawn A. Laney, MS, CGC, CCRC (Chair)
Genetic Counselor and Assistant Professor
Emory University School of Medicine, Emory University
Atlanta, GA, USA

Dawn A. Laney is a genetic counselor, assistant professor, clinical researcher, and program leader at the Lysosomal Storage Disease Center, and Director of the Emory Genetic Clinical Trials Center in the Department of Human Genetics, both at Emory University in Atlanta, GA, USA. She earned her Master’s degree in medical genetics from Sarah Lawrence College in 1999, followed by certification from the American Board of Genetic Counseling in 2002.


Ms Laney’s clinical and research interests focus on Fabry disease. Since joining the Emory Division of Medical Genetics in 2002, she has been involved in the follow-up of abnormal metabolic newborn screening for the whole state of Georgia, the development, and management of a genetic infusion center, and ysosomal storage disease clinical care and research. She is a cofounder of ThinkGenetic, Inc., which empowers patients with information about possible genetic causes for their medical issues and provides real-life answers to their questions about the impact of living with a genetic disease. Ms Laney enjoys writing children’s books, most of which are about living with Fabry disease.

Robert J. Hopkin, MD, FACMG
Clinical Geneticist, Division of Human Genetics
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH, USA

Robert J. Hopkin is Associate Professor of Clinical Pediatrics at Cincinnati Children’s Hospital Medical Center in Cincinnati, OH, USA. Dr Hopkin graduated from the University of Nevada Medical School in 1990. He completed his residency and chief residency in Pediatrics at the Phoenix Children’s Hospital, Maricopa Medical Center Pediatrics Residency Program in 1994, then completing his training in Medical Genetics at Cincinnati Children’s Hospital Medical Center in 1997.

Dr Hopkin has worked in the field of lysosomal diseases for more than 25 years, participating in clinical trials and other clinical research on Fabry disease, Pompe disease, Hunter syndrome, and other disorders. Most of his time is spent caring for adult and pediatric patients with genetic disorders. As the Director of the Medical Genetics Residency and Fellowship Program at Cincinnati Children’s Hospital, he is also actively involved in the education of healthcare providers on the application of genetics for patient care. The unifying principle in his research interests is application of scientific knowledge to improve outcomes for patients afflicted with genetic disorders.

Anjay Rastogi, MD, PhD
Professor and Clinical Chief of Nephrology
UCLA David Geffen School of Medicine
Los Angeles, CA, USA

Anjay Rastogi is Professor of Nephrology and Clinical Chief of Nephrology at the David Geffen School of Medicine at University College California (UCLA) in Los Angeles, CA, USA. He is board certified in nephrology and has a doctoral degree (PhD) in pharmacology. Dr Rastogi completed an internal medicine residency and nephrology fellowship at UCLA, where he also completed his graduate training under the mentorship of Nobel Laureate Professor, Louis Ignarro.

Dr Rastogi founded, and is the director of, the UCLA CORE Kidney Health Program; this includes the Circle of CORE, a patient advocacy and support group formed by patients that includes kidney recipients and living kidney donors. He also serves as the director of many other programs, including the Genetics Program at UCLA, all of which are dedicated to providing the best comprehensive and integrated care to patients and their families. Dr Rastogi was awarded 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.

Disclosures of conflicts of interest
Postgraduate Institute for Medicine (PIM) requires instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflict of interest (COI) they may have as related to the content of this activity. All identified COI are thoroughly vetted and resolved according to PIM policy. PIM is committed to providing its learners with high quality activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest.


Dawn A. Laney, MS, CGC, CCRC
Consulting fees from Spark Therapeutics, Sanofi Genzyme, Amicus Therapeutics. Ownership interest (less than 5%) in ThinkGenetic, Inc.

Robert J. Hopkin, MD, FACMG
Consulting fees from Alexion Pharmaceuticals Inc., Amicus Therapeutics, AVROBIO, Inc., Chiesi USA, Inc., Sangamo Therapeutics, Inc., Sanofi Genzyme, Takeda. Advisory arrangements with Alexion Pharmaceuticals Inc., Amicus Therapeutics, Sanofi Genzyme. Speakers bureau for Alexion Pharmaceuticals Inc., Sanofi Genzyme. Grants/contracts research from Alexion Pharmaceuticals Inc., Amicus Therapeutics, Idorsia Pharmaceuticals Ltd, Protalix Biotherapeutics, Sanofi Genzyme, Takeda.

Anjay Rastogi, MD, PhD
Consulting fees from Adept Field Solutions, Spire Learning, Sanofi.

Planners and managers
The PIM planners and managers have nothing to disclose. The Ology Medical Education planners and managers have nothing to disclose.

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.

Joint accreditation statement
In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and Ology Medical Education. Postgraduate Institute for Medicine 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.

Physician continuing medical education
The Postgraduate Institute for Medicine designates this enduring material for a maximum of up to 1.5 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

ACMG will provide credit for genetic counselors (NSGC) and ASCLS (Professional Acknowledgment for Continuing Education (P.A.C.E.®).

A statement of credit will be issued only upon receipt of a completed evaluation form.

This activity is supported by an independent educational grant from Amicus Therapeutics Inc.

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 patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities. The course materials are for educational purposes only and should not be reproduced or distributed in any way. If you wish to reproduce, store in a retrieval system, transmit in any form or by any means, electronic, mechanical, photocopying, recording, or otherwise, any part of the material presented, you will need to obtain all the necessary permissions by writing to the publisher, the original author, or any other current copyright owner.

If you have attended this satellite symposium and would like to receive your CME credits, please fill out the evaluation form.

A multidisciplinary conversation on the stories of women with Fabry disease

CME accredited hybrid satellite symposium

This activity is supported by an independent educational grant from Amicus Therapeutics Inc.

Register for ACMG 2022

Registration is obligatory for access to this satellite symposium, and is part of the general congress registration; please register for both the satellite symposium and the congress.


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