Although all kinds of rare diseases taken together have an enormous impact, there is a significant gap between basic research and clinical interventions. Opportunities now exist to accelerate drug development for the treatment of rare diseases. Disease foundations and research centers worldwide focus on better understanding rare disorders. However, developing drugs for a rare disease can be challenging due to specific rare disease characteristics.
The challenges include: Rare diseases are usually genetic diseases; hence, employing pharmacogenetics to develop treatments and using whole genome sequencing to identify the etiologies for such diseases are appropriate strategies to exploit. However, the funds required for the research are massive. Beginning with high throughput screening of small molecules, the benefits and challenges of target-based and phenotypic screens are required. Drug…
