Friday, January 10th, 2020
New York City

Friday, January 10th, 2020

Conrad New York Hotel

Studio 6 Room
102 N. End Avenue
New York, NY

Fairmont Olympic Hotel

Congress Room
411 University Street
Seattle, WA

Friday, January 17th, 2020

Friday, January 17th, 2020
Washington, DC

Hotel Fairmont Chicago Millennium Park

State Room
200 N. Columbus Drive
Chicago, IL

Capital Hilton Hotel

Statler A/B Room
1001 16 th Street NW
Washington, DC

Friday, January 24th, 2020

Friday, January 24th, 2020
San Francisco

Ritz Carlton Hotel

Maple Room
2121 McKinney Avenue
Dallas, TX

Parc 55 Hotel

Embarcadero Room
55 Cyril Magnin Street
San Francisco, CA

Registration/Buffet Dinner: 6:00 PM
Scientific Session: 6:30 PM


6:00 PM

Registration / Buffet Dinner

6:25 PM

Welcome, Disclosures, Acknowledgements

6:30 PM

Clinical Signs and Symptoms
Multiple Organ Involvement
Hematologic Co-Morbidities
Testing for ASMD
Treatment Options

7:05 PM

NIEMANN PICK TYPE A AND TYPE B (or Acid sphingomyelinase Deficiency (ASMD)
Epidemiology and Clinical Presentations
The Metabolic Defect
Disease Manifestations
Treatment Plans

7:40 PM

Closing Comments, Audience Q&A

8:00 PM




Margaret M. McGovern, MD, PhD
Knapp Professor of Pediatrics
Dean for Clinical Affairs
Stony Brook University School of Medicine
Stony Brook, NY

Seymour Packman, MD
Professor Emeritus
Division of Medical Genetics / Department of Pediatrics
University of California San Francisco
San Francisco, CA



Paige Kaplan, MBBCh
Professor Emerita
Perelman School of Medicine - University of Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, PA

Amy Yang, MD
Assistant Professor
Department of Molecular and Medical Genetics
Oregon Health & Science University
Portland, OR

Pamela Arn, MD
Operational Vice President of Education
Nemours Children's Health System
Interim Medical Director of Precision Medicine
Special Consultant, Department of Clinical Genomics
Mayo Clinic
Jacksonville, FL

Jeremy Lorber, MD
Cedars-Sinai Tower Hematology Oncology Medical Group
Beverly Hills, CA



EXCEL Continuing Education is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

EXCEL Continuing Education designates this live activity for a maximum of 1.5 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.


At the conclusion of this conference, you should be better able to:

Summarize the epidemiology and clinical manifestations of Gaucher disease and ASMD to better understand these diseases

Discuss metabolic defect in Gaucher disease and ASMD and how this affects multiple organs

Explain the association of Gaucher disease to hematologic co-morbidities

Discuss hematologic manifestations that often present in ASMD and why patients suspected of having Gaucher disease should also be tested for ASMD

Review treatment options including enzyme replacement therapy, gene therapy, and bone marrow transplantation







EXCEL CE complies with the Americans Disabilities Act. If any participant is in need of reasonable assistance, please FAX a written request to 305-598-7252 for consideration at least 2 weeks prior to the activity.