The safety and efficacy of these products for the indication under study have not yet been established by the relevant regulatory authorities.
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Hepatorenal Syndrome Type 1 (HRS-1) is a life-threatening condition that consists of rapid deterioration in kidney function in individuals with cirrhosis or liver failure. HRS-1 affects from 10,000 to as many as 30,000 patients in the U.S. This condition can lead to multi-organ failure including acute kidney failure, and is often fatal unless a liver transplant is performed or the condition is reversed. There is no FDA-approved pharmacological therapy for treatment of HRS-1 in the U.S.
Reversal of HRS has been shown to increase survival and improve clinical outcomes. Terlipressin is the most widely studied and clinically accepted pharmacological therapy globally for patients with HRS-1.
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Full thickness burns (also known as third degree burns) affect all layers of the skin, and may cause damage down to underlying muscles. These burns carry severe risk for complications, such as infections, blood loss and shock, which could lead to organ failure or death. The current standard of care for severe burns is skin-grafting, or autografting, which is the surgical harvest of healthy skin from an uninjured site on the patient’s body followed by transplant to the wound. It results in a donor site wound that requires medical management of pain, and may result in infection and/or scarring.
A previous study in full thickness skin defects showed that StrataGraft skin tissue remained intact and viable throughout the seven-day placement period when it was used as a temporary cover to prepare the wound for skin-grafting. StrataGraft skin tissue is being studied further as a way to reduce the amount of autografting required in these patients.
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The complement cascade is a group of proteins that are involved in the immune system and that play a role in the development of inflammation. These proteins are known to contribute to the pathogenesis of many diseases, including autoimmune diseases.
The RNAi drug target is designed to inhibit the complement cascade.
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The complement cascade is a group of proteins that are involved in the immune system and that play a role in the development of inflammation. These proteins are known to contribute to the pathogenesis of many diseases, including autoimmune diseases.
The RNAi drug target is designed to inhibit the complement cascade.
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The complement cascade is a group of proteins that are involved in the immune system and that play a role in the development of inflammation. These proteins are known to contribute to the pathogenesis of many diseases, including autoimmune diseases.
The RNAi drug target is designed to inhibit the complement cascade.
FT: Full Thickness, HRS: Hepatorenal Syndrome, RNAi: Ribonecleic Acid interference
*Collaboration with Silence Therapeutics