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Rare Disease Clinical Trials

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Overview / Abstract:

When caring for an individual with a rare disease who has exhausted standard treatments or for whom there are no treatments available, exploration of clinical trials, and possibly expanded access programs, may be warranted. However, many care team members and their patients may not know where to begin with these options. In addition, a number of barriers to participation may exist for both the patient and the care team. Clinicians caring for people with rare diseases, therefore, would benefit from education about how to find appropriate trials for their patients with rare diseases, and how to overcome patient and team barriers to participation.


Nov 30, 2020


Nurse Practitioner , Nursing CNE, Physician CME, Physician Assistant CME


Online, Webinar / Webcast / Video

Credits / Hours



American Academy of CME, Inc.

Presenters / Authors / Faculty

Heather Lau, MD, MS, NYU School of Medicine, New York, NY

Morie A Gertz, MD, MACP, Professor of Medicine, Mayo Clinic, Rochester, MN

Timothy Craig, DO, Penn State University, Hershey, PA

Eric T. Wong, MD, FANA, FAAN, Harvard Medical School, Boston, MA

Activity Specialities / Related Topics

Research, Pulmonary Medicine / COPD, Pediatrics, Oncology / Cancer / Radiation Therapy, Neurology, Nephrology, Internal Medicine, Hematology, Geriatrics / LTC, Education / Teaching

Sponsors / Supporters / Grant Providers

Sanofi Genzyme, Grifols, Novocure, and Ultragenyx Pharmaceuticals

Keywords / Search Terms

CheckRare Rare Disease, Clinical Trials, Lysosomal Storage Diseases, Gaucher, Fabry, MPS, Pulmonology Clinical Trials, Rare Cancers, Rare Blood Disorders, Rare Pulmonary diseases, Harvard, NYU, Free CE CME, rare disease, NYU School of Medicine, Heather Lau MD, Morie A. Gertz MD, Rochester Mayo Clinic, Timothy Craig DO, Allergy and Immunology, Penn State University, Eric T Wong MD, Harvard Medical School, Mucopolysaccharidoses

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