Comprehensive public-private partnerships, the National Institutes of Health (NIH) and rare diseases community, which is sighted a renewed industry interest in markets, have resulted in an increase of interventions for rare diseases. There are now involving 4,770 progressing research projects, barring clinical trials, covers numerous diseases and research on this area requires multidisciplinary approach in order to determine innovative treatments for the initial diagnosis and treatment procedures. Requirements of the pediatric populace with rare diseases autonomously organizing from that of grown-ups. Since about 50 to 75% of the rare diseases begin in childhood, these pediatric disorders deserve special importance. The expanding role and outreach activities of patient-advocacy groups have increased public awareness.