JAAPA CME Post-Test November 2024

Novel Gene Therapies for Sickle Cell Disease, Duchenne Muscular Dystrophy, and Hemophilia A

This activity discusses novel genetic therapies for sickle cell disease, Duchenne muscular dystrophy, and hemophilia A. Gene therapies have the potential to deliver more targeted and effective approaches to treatment, especially for rare diseases for which the availability of approved therapies is limited. This activity describes the first FDA-approved CRISPR/Cas9 treatment and the treatment protocols, indications, warnings, precautions, cost, and contraindications of four novel genetic therapies.

Learning Objectives

At the conclusion of this activity, participants should be able to:
  • Compare various recent FDA-approved genetic therapies in terms of complexity, time duration, and adverse reactions
  • Describe how CRISPR/Cas9 is used in gene editing therapies
  • Discuss the mechanism of action of gene editing therapies
  • Outline different genetic diseases that have positive outcomes with gene editing treatment including sickle cell disease, Duchenne muscular dystrophy, and hemophilia A

Polycystic Ovary Syndrome: Fertility, Prenatal Care, Pregnancy, and Postpartum Care

Polycystic ovary syndrome (PCOS) is a complex, heterogeneous reproductive endocrinopathy affecting 8% to 13% of women in their reproductive years. PCOS has a vast array of clinical manifestations, including metabolic complications, biochemical and phenotypical hyperandrogenism, menstrual cycle dysfunction, and anovulation resulting in infertility. Understanding the relationship of the disorder’s pathophysiologic and clinical manifestations is key to successfully managing patients with PCOS. Patients may have different prenatal, perinatal, and postnatal experiences than those who do not have PCOS, and may require a higher level of care or treatment plan adjustments.
 

Learning Objectives

At the conclusion of this activity, participants should be able to:
  • Define PCOS and its clinical manifestations
  • Define the Rotterdam criteria for diagnosing PCOS and understand varying presentations of PCOS based on phenotype
  • Outline treatment options for patients with PCOS and identify the clinical manifestations these treatments may improve
  • Explain the role of optimal PCOS management on fertility and pregnancy
  • Outline the differences in treatment management and complicating factors during prenatal, perinatal, and postpartum care for patients with PCOS
 

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