Common problems for patients

Common Problems for patients and families of patients with rare and ultra-rare diseases are:

  • medical problemsLack of access to correct diagnosis;
  • Delay in diagnosis, sometimes decades;
  • Lack of quality information on the disease;
  • Lack of scientific knowledge of the disease;
  • Heavy social consequences for patients and their families and caregivers;
  • Lack of appropriate quality healthcare;
  • Inequities and difficulties in access to treatment and care;
  • Lack of political support because the rare disease community is fragmented;
  • Ignored in policy making;
  • Huge cost or fights to get orphan drugs used to treat symptoms to some extent;
  • It is possible that every medical professional you see will never have treated the disease before.