Sickle cell disease (SCD) is a group of inherited rare blood disorders that affects less than 1:2000 births in the United States.
Cystic fibrosis (CF) is an inherited disorder that affects 1:3500 white people of Northern European descent in the United States.
Kawasaki disease is the number one acquired heart disease in children in the United States and Japan; 6000 are diagnosed with the disease per year, a vast majority of patients are children between 1 to 4 years old, and 25% of patients develop coronary artery aneurysms, a fatal heart disease, as a result when not caught by diagnosis.
Men, women, and children living with rare diseases often face uncertainty and hardship when navigating the healthcare system. Care coordination, next step diagnostics, vetted resources, and relevant clinical tools are critical needs for patients and health teams to provide equal access to healthcare services for patients affected by rare diseases.
Patients with rare diseases and unmet needs deserve a strong voice in direct medical care …
