An Orphan Drug receives an Orphan Drug Designation from the US FDA and the European Medical Agency. In the US, rare genetic diseases such as Tay-Sachs and Gaucher Disease are designated as Orphan Diseases because they have fewer than 200,000 patients in the US. The FDA created the Orphan Drug act in order to compel the pharmaceutical and biotechnology industries to develop drugs for small patient groups. Orphan Diseases and Orphan Drugs can be attributed to as few as 25 or 50 patients.
ERAD Therapeutics Inc.
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WHO WE ARE
- Private, pre-clinical gene therapy and orphan drug company
- Experienced Senior Management team and Board of Directors.
- On-going collaborations with Key stakeholders in Range Genetic Diseases.
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WHAT WE DO
- With an initial focus on neuromuscular and lysosomal storage diseases, we develop novel gene therapies to treat rare genetic diseases.
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OUR APPROACH
- We are taking a methodical approach to targeted gene therapies as well as a novel gene therapy-based approach to a broad spectrum of rare genetic diseases.
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WHY OUR CANDIDATES
- Few if any therapies exist to address the neurological aspects of rare genetic diseases.
- Our candidates are capable of being delivered across the blood brain barrier.
- Our therapeutic platforms will potentially address a multitude of rare genetic diseases.
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NEUROMUSCULAR DISEASE
Neuromuscular diseases are a large group of disorders that primarily disrupt the normal functioning of muscles and the nerves that control them. This leads to muscle weakness, impaired movement, and often progressive disability.
Examples of neuromuscular diseases are:
- Muscular Dystrophy including Duchenne, Becker and many more;
- Charcot- Marie-Tooth Disease and other neuropathies;
- Myesthenia-gravis and more
GAUCHER DISEASE
Gaucher Disease is the most common Lysosomal Storage Disease, yet it is still considered an Orphan Disease. Currently affecting over 8,000 American’s, it is a genetic disorder resulting in an enzyme deficiency. Type 1 disease usually results in an enlarged liver, enlarged spleen, anemia and bone disease. Without therapy this significantly impacts both the quality and duration of life. Type 3 disease impacts all of the above as well as causing progressive and ultimately fatal neurological deterioration.