Rare Disease Information

Challenges Faced by Rare Disease Patients and Families

1. Delayed or Missed Diagnoses

   • On average, it takes 4-8 years to receive a correct diagnosis for a rare disease.

   • Misdiagnoses can lead to unnecessary treatments and prolonged suffering.

2. Limited Treatment Options

   • Only 5% of rare diseases have FDA-approved treatments.

   • Patients often rely on experimental therapies or clinical trials for care.

3. Emotional and Financial Burdens

   • Rare diseases often require lifelong management, creating emotional and financial stress for patients and their families.

   • Insurance may not fully cover specialized treatments or experimental care.