Timely diagnosis of Sanfilippo syndrome (MPS III)
December 29, 2020
August 29, 2021
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Jointly provided by Postgraduate Institute for Medicine, Cure Sanfilippo Foundation, and P2P Syncro.
This activity is supported in part by an independent education grant from Abeona Therapeutics.
- Physicians — maximum of 0.50 AMA PRA Category 1 Credit(s)™
All other health care professionals completing this course will be issued a statement of participation.
This activity is intended for pediatricians in clinical practice in the US.
Statement of Need/Program Overview
What To Learn:
- Sanfilippo is in the differentials of developmental delay, autism, and ADHD
- Diagnostic testing can be initiated by primary care pediatricians
Upon completion of this activity, participants should be better able to:
- Review Sanfilippo syndrome (MPS III) is a lysosomal storage disease
- Relate the progressive nature of the disease
- Describe clinical features frequently present at the time of diagnosis
- Name three common pediatric diagnoses associated with Sanfilippo syndrome
- Consider ordering urine GAG test and/or MPS enzyme panel once clinical suspicion is raised
Cara O'Neill, MD
Dr. O’Neill is a pediatrician and the Chief Science Officer at Cure Sanfilippo Foundation. She works closely with patients, providers and scientists in the field of Sanfilippo syndrome on a number of collaborative initiatives including the development of innovative clinical trial outcome measures, caregiver preference research, and trial design for rare neurologic disease. She has also spearheaded the initiative of developing clinical management guidelines for Sanfilippo syndrome.
Eloise Prijoles, MD
Dr. Prijoles is board certified in pediatrics and medical genetics. She received her genetics training at Stanford University and currently provides clinical genetic services through the Greenwood Genetic Center in South Carolina. She has more than a decade of experience of providing care for patients with inherited disorders and known or suspected genetic abnormalities.
In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and P2P Syncro. 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.
The Postgraduate Institute for Medicine designates this enduring material for a maximum of 0.5 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
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. Dr. Cara O’Neill received fees for non-CME/CE services from Lysogene. Dr. Eloise Prijoles has nothing to disclose. The PIM planners and managers have nothing to disclose. The P2P Syncro planners and managers have nothing to disclose.
Instructions for Participation and Credit
There are no fees for participating and receiving CME credit for this enduring activity. To receive CME credit participants must:
- Read the CME/CE information and faculty disclosures.
- Participate in the online activity.
- Submit the evaluation form.
Course Viewing Requirements
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above
Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above
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.
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.
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