Rare diseases are an important public-health issue and a challenge for the medical community. Most rare diseases are genetic disorders, which are often severely disabling, substantially affect life expectancy, and impair physical and mental abilities. Currently, there are about 7,000 identified rare diseases, together affecting 10% of the population. However, fewer than 6% of all rare diseases have an approved treatment option, highlighting their tremendous unmet needs in drug development. The process of repurposing drugs for new indications, compared with the development of novel orphan drugs, is a time-saving and cost-efficient method resulting in higher success rates, which can therefore drastically reduce the risk of drug development for rare diseases.

MCE carefully collects a unique of 1747 compounds studied in preclinical, clinical trials or approved used in rare diseases treatment. MCE rare diseases drug library is a useful tool for the research of rare diseases. All compounds can provide corresponding indications for rare diseases.

For pre-dissolved solutions, 10 mM for compounds with the solubility not lower than 10 mM, 2 mM for compounds with solubility between 2 mM and 10 mM, and 3 mg/mL for compounds with unconfirmed molecular weight and solubility not lower than 3 mg/mL.