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An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The ...
The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome or muscular dystrophy which affect small numbers of individuals residing in the United States.
This was the first count since 1983, demonstrating that there were >10,500 rare diseases where prior estimates had been ~7,000 in the Orphan Drug Act. [5] Global Genes has also estimated that currently approximately 10,000 rare diseases exist globally, with 80% of these having identified genetic origins.
The FDA grants orphan drug and rare pediatric disease designations to Ionis' (IONS) ION582 for treating Angelman syndrome. These designations will help incentivize development of the candidate.
AVG-002 and AVG-003 are potentially first-in-class disease-modifying therapies for newborns with rare lethal Surfactant Protein B (SP-B) and ABCA-3 deficiencies, respectively. AVG-002 is being developed under Rare Pediatric Disease Designation and Orphan Drug Designation with preparations for its clinical development underway.
(Reuters) -X4 Pharmaceuticals said on Monday the U.S. FDA has approved its therapy to treat a rare genetic immunodeficiency disease in patients 12 years of age and older. X4's mavorixafor, to be ...
There are more than 6,000 known rare diseases, and it is estimated that about 25 million Americans are affected by them (as of 2002). [2] Prior to the RDA was the Orphan Drug Act of 1983, which was designed to facilitate the development and commercialization of drugs to treat rare diseases, termed orphan drugs. This act, however, did not ...
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