Ralf Rosskamp, M.D., Chief Medical Director at Dicerna Pharmaceuticals discusses the latest clinical data evaluating nedosiran as a treatment option for primary hyperoxaluria (PH).

PH is a family of rare disorders characterized by recurrent kidney and bladder stones. It is the result of an overproduction of oxalate, which can combine with calcium to form calcium oxalate and subsequently kidney and bladder stones. These stones cause damage to the kidneys among other organs, and increase the chance of hematuria, urinary tract infections, and end-stage renal disease (ESRD). 

Three gene mutations have been identified to cause the three known types of PH: AGXT mutations cause PH type 1 (PH1), GRHPR mutations cause PH type 2 (PH2), and HOGA1 mutations cause PH type 3 (PH3).

In this interview, Dr. Rosskamp summarized the result of their proof of concept study that showed nedosiran to be safe and effective.

An ongoing open-label extension is currently underway.

For more information about PH and other rare kidney diseases, visit checkrare.com/diseases/kidney

 

 

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