Living With Rare Diseases
Rare diseases can be devastating to live with, and can be difficult for even the few physicians who are familiar with them to diagnose, so even getting an accurate diagnosis becomes a process of missed opportunities and delays.
Learning you have a rare disease can be one of the most difficult diagnoses to receive. When you do get the right diagnosis, few, if any, therapies are available, and often research or regulatory pathways are unclear.
Every day, people living with rare diseases, their caregivers, and families face fears of the unknown with courage, tenacity, and grace that inspire us to continue to strive to do everything we can to transform their lives.
Rare Diseases: A Massive Challenge That's Hard to See
LIVING WITH HPP
KNOWN RARE DISEASES IN THE WORLD
HAVE APPROVED TREATMENT OPTIONS
OF PEOPLE LIVING WITH RARE DISEASES ARE CHILDREN
Patient Experience Team
Our global Patient Experience team is responsible for ensuring that patient perspectives and engagement with the rare disease community are always at the forefront of our work. We seek to understand and incorporate insights from people living with rare diseases and their caregivers into our development plans, medicines, and services. Our promise is to partner with the rare disease communities we support to better understand the journeys patients experience in their search for diagnosis, treatment, disease education, and management of their conditions. We do this through working closely with advocacy organizations and through direct patient engagements to realize shared ambitions while respecting health information privacy.
Every Day Strengthens Our Impact
Our approach to serving patients with rare diseases focuses on disease awareness, diagnostic initiatives, and patient support. Every patient matters, and we hold ourselves accountable to serving each patient, and their family, every step of the way.
When I hold Adelyn now, it’s like holding the sun––a ball of warmth. She just looks at you and warms your heart.”JOE, ADELYN'S DAD LIVING WITH HPP