Orphan diseases and Public Health

The field of rare diseases is difficult and heterogeneous and suffers from a deficit of scientific and medical knowledge .The landscape of rare disease is constant evolving the new rare diseases and conditions being reported and identified regularly in medical literature. Apart from a few rare diseases where significant progress has been made, still the field is nascent stage. For a long time, doctors, researchers and policy makers were unaware of rare diseases and until very recently no real research or public health policy concerning issues related to the field .This posses challenges in development of comprehended policy on rare diseases. Nevertheless, it is important to take step, in the short as well as long term, with the objective of talking rare diseases in a comprehensive and holistic manner.

Awareness of rare diseases

Public health care

Challenges in public health

Cystic fibrosis

Process of gene therapy

Awareness of rare diseases