A review on the rare diseases and subsequent orphan drugs

Abstract

Worldwide among every existing disease, approximately 5000-8000 are often referred to as rare diseases due to their uncommon, chronic, progressive, degenerative, and life-threatening characteristics. This review specifically deals with Duchenne Muscular Dystrophy, Friedreich's Ataxia, and Transthyretin Amyloid Polyneuropathy; three rare conditions each with the scope of better orphan treatment opportunities. In spite of some orphan drugs’ improving results for treating each rare disease, it has been however recommended that only a precise and effective orphan treatment can provide optimal life. In response to these rare diseases, substantial cooperation among the stakeholders, funders, orphan drug developers, regulatory authorities, policymakers, governments, and immense global effort is crucial. This review paper is hence a nuanced analysis of ongoing strategies to patently identify rare diseases, three specific rare diseases profile along with their impending orphan drug candidates, and their pre-and post-development challenges.