NEEDS STATEMENT

TANGO2 deficiency disorder (TDD) is a serious multisystem genetic disease that can cause life threatening metabolic crises and cardiac arrhythmias. Currently, clinicians encounter complex TDD patients in various in and outpatient settings (i.e. specialty practices, ED, and primary care). Because of a lack of knowledge, training, clinical encounters, published literature and ongoing research exposure, many TDD patients are improperly assessed at a critical juncture in their existence. This disorder causes significant morbidity in children, primarily affecting neurodevelopment & cardiovascular health. About 20% of those with TDD pass away by age 18 due to severe complications like rhabdomyolysis & difficult to treat cardiac arrhythmias triggered by fasting, dehydration, infection & viral illnesses. Therefore, patient outcomes can be greatly improved as research advances and the body of scientific knowledge continuously broadens.

The activity plans to address the educational needs that underlie the professional practice gaps of the learners by providing learners with a comprehensive symposium agenda that highlights current research initiatives that support scientific advances in TDD coupled with recent trends in TDD clinical care advances, including recommendations for TDD therapeutics in the future.

TARGET AUDIENCE

This activity is intended for Primary Care, Cardiology, Neurology, Genetics, Nutrition, Pediatrics, residents, fellows, nurses, postdoc fellows, students, Genomics, pharmacy, immunology, allergy, molecular and cell biology, movement disorders, speech and language, Metabolomics and TDD families.

EDUCATIONAL OBJECTIVES

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

• Describe the current TANGO2 deficiency disorder research landscape and its critical relevance.
• Evaluate the clinical recommendations and pertinent published literature on TANGO2 deficiency disorder to assess its current understanding and diagnostic approaches.
• Evaluate and identify research and clinical experts in TANGO2 deficiency disorder for the purpose of facilitating consults and referrals related to this genetic condition.

EDUCATIONAL METHODS

Lecture

EVALUATION

Evaluation by questionnaire will address program content, presentation, and possible bias.

PROGRAM COORDINATOR

Deena Chisholm, MPH, CHES - deena.chisholm@tango2research.org

COURSE DIRECTOR 

Lilei Zhang, MD, PHD - Lilei.Zhang@bcm.edu