There are two major problems with the development of therapies for rare diseases. First,
among over 7000 such diseases, the vast majority are caused by genetic defects and/or …

Background and aims: Orphan diseases, or rare diseases, are defined in Europe as
diseases that affect less than 5 out of every 10,000 citizens. Given the small number of cases …

The author discusses the rising prominence of orphan drugs for treating rare disease
defined as one that affects less than 200,000 persons in the US and less than 5 in 10,000 …

A great number of people suffer from obscure and unknown diseases for which there have
been few attempts to create treatments prior to the Orphan Drug Act of 1983. Today, the Act …

Editorial collaboration and coordination between funding institutes to make joint efforts
possible and achieve maximum efficiency of funding structures. Currently, IRDiRC is …

Rare diseases and orphan drugs represent an exciting and growing field at the conjunction
of many disciplines of science, from basic chemistry, biology, and physiology to medicine …

Historically, high costs associated with bringing a drug to market and limited potential for
revenue discouraged sponsors from developing products for diseases impacting small …

Not only in Europe and USA, but also in many other countries rare disorders—so-called
orphan diseases—have attracted more and more attention. The formation of specialized …

Orphan drugs are the stepchildren of the pharmaceutical industry. These medicinal products
that are used primarily for the prevention or treatment of life‑threatening or debilitating rare …

Many neurological diseases or conditions are rare disorders. The Orphan Drug Act (ODA) of
1983 was promulgated to promote the development of products for such conditions. In this …