This activity will explore the most recent advances of this rapidly evolving therapy, including current data on its efficacy, limitations and challenges in the treatment of multiple myeloma and solid tumors. It will provide an overview of currently existing guidelines for CAR T-cell therapy and will describe best practices in determining individuals’ eligibility for this type of therapy, including factors that may affect treatment efficacy and outcomes.

This session will examine the genetic approaches being explored for these diseases, such as the introduction of a corrected or modified β-globin gene and gene-sequence editing. It will also explore the genetic modalities utilized, such as genome-editing technologies including the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene-editing system and lentiviral vectors. The safety, efficacy and challenges associated with these therapies will also be outlined.

This activity will review the symptoms and signs of SM, provide an overview of new and current therapies and identify SM guideline updates. The value of an interdisciplinary team–based approach along the continuum of care for affected individuals will also be discussed as a definitive step toward optimizing outcomes. 

In this podcast Nealie White, an individual with Pomp disease, will share her insights and perspectives, and panelists will provide strategies that the interprofessional health care team can utilize to better support people who are impacted by rare diseases.