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Reaching Milestones in ENPP1 Deficiency: Advances in Treatment

Reaching Milestones in ENPP1 Deficiency: Advances in Treatment

Friday, September 05, 2025

CME Satellite Symposium at the ASBMR, Seattle Convention Center

705 Pike Street

Seattle, WA 98101

To attend, please ensure you are registered for the ASBMR annual meeting. 

Generalized Arterial Calcification of Infancy (GACI) is a rare autosomal recessive disorder associated with pathogenic variants in ENPP1, the major gene involved in this condition, and in ABCC6, which is involved in a small fraction of affected individuals. GACI is initially characterized by an abnormal ectopic mineralization process in arteries and soft tissue, causing critical blockages which can reduce blood flow to organs which in turn can cause strokes, heart attacks, and death.

In this live in-person CME-accredited satellite symposium, two rare mineralization disorder experts will discuss the natural history of ENPP1 deficiency including GACI, assess pre-clinical trial data, and end the presentation with a discussion of emerging treatment options that have the potential to improve the outcomes of patients with this disorder.

Schedule

* Times are PST
12:30 PM Registration and Boxed Lunches
12:40 PM Welcome, Disclosures, Supporter Acknowledgement
12:45 PM Presentation

ENPP1 Deficiency: Natural History of Disease
Clinical Presentations: Diagnosis and Ongoing Lifelong Management Strategies
GACI: Pre-clinical Trial Data and Emerging Treatment Options

01:30 PM Closing Comments, Audience Q&A
01:45 PM Adjourn

Presenters

Robert A. Wermers, MD

Professor of Medicine and Chair
Division of Endocrinology, Diabetes, Metabolism, and Nutrition
Mayo Clinic
Rochester, MN

Agnès Linglart, MD

Professor
Pediatric Endocrinologist and Professor of Pediatrics
Head, French National Center of Reference
Bicêtre APHP Hospital
Bicêtre, France

Accreditation

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 1.0 AMA PRA Category 1 Credits. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Canadian CME: Through an agreement between the ACCME and the Royal College of Physicians and Surgeons of Canada, medical practitioners participating in the Royal College MOC Program may record completion of accredited activities registered under the ACCME’s “CME in Support of MOC” program in Section 3 of the Royal College’s MOC Program.

The UEMS-EACCME®: European Union of Medical Specialists and European Accreditation Council for CME have mutual recognition agreements with the American Medical Association for live events and e-learning materials. European physicians can earn their ECMEC®s (European CME credits) by either attending a live presentation approved by an ACCME accredited provider such as EXCEL or participating in e-learning activities (on-demand webcasts) from accredited providers from the USA that have been certified for AMA PRA Category 1 Credits™.

Physician assistants, nurse practitioners, and nurses may participate in this educational activity and earn a certificate of completion as AAPA, AANP, and ANCC accept AMA PRA Category 1 Credits TM through their reciprocity agreements.

Educational Objectives

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

 

  • Discuss the role of ENPPI enzyme in skeletal mineralization
  • Recognize the clinical presentation of ENPPI deficiency across the lifespan while discussing diagnostic and lifelong management strategies
  • Assess the therapeutic potential of emerging replacement therapy for the treatment of ENPPI deficiency

Supporter

INOZYME PHARMA