Medical – Hereditary Angioedema (HAE):

Defining overall disease burden for patients, families and others impacted by HAE/ bradykinin-mediated angioedema

  • Understanding the impact of HAE disease and unmet needs of underrepresented patient populations
  • Understanding HAE disease and treatment burden on quality of life (including physical and mental health impacts) on patients and caregivers
  • Healthcare utilization costs and financial burden of HAE
  • Evolution of understanding and classification of bradykinin-mediated angioedemas (i.e., HAE normal C1-INH, hereditary, acquired, contact pathway mutations)

Redefining HAE/bradykinin-mediated angioedema disease management goals and optimal outcomes

  • Diagnosis, management, and outcome measures of success for patients living with HAE/bradykinin-mediated angioedema
  • Review of treatment guidelines/recommendations
  • Individualized support and shared decision making to improve patient experience and long-term goals for success
    • Options on switching from previous prophylaxis, on-demand treatment, and patient-specific considerations for approved therapies (individualized approach)
  • Diagnosis and management of pediatric patients with HAE/bradykinin-mediated angioedema

Patient Advocacy:

Local, regional, and global patient advocacy focused on:

  • Hereditary Angioedema (HAE)
  • Diabetic Macular Edema (DME) and vision loss
  • Netherton Syndrome
  • Broader rare disease community