Is Hope on the Horizon for a Primary Hyperoxaluria Type 1 Cure?

This past July, professionals, patients, and families with loved ones who struggle with primary hyperoxaluria Type 1, 2 and 3 were offered a glimpse of hope and a better future. When leading pharmaceutical companies presented a host of new treatments, the attendees were all ears, according to an online article in Rare Disease Report.

Primary hyperoxaluria is a collection of rare, genetic liver disorders that cause a buildup of oxalate. Oxalate is usually removed from the body by the kidneys, but when high amounts of oxalate start accumulating in the body, the result can be renal and bladder stones. Current treatments typically involve drinking excessive amounts of fluids, avoiding vitamins C and D, and medication therapies. To learn more about primary hyperoxaluria, click here.

There are three types of primary hyperoxaluria.

  1. Type 1 – Most severe, most common, rapidly progressive, 50 percent develop end-stage renal disease
  2. Type 2 – Lower elevated levels of oxalate, 25 percent develop end-stage renal disease
  3. Type 3 – Most rare, patients typically don’t progress to end-stage renal disease

The workshop was hosted by the Oxalosis and Hyperoxaluria Foundation (OHF) in Spain. Interest ran high when the pharmaceutical companies took the main stage to present products and a number of possible medications that were in development. The products are online to be tested on people with primary hyperoxaluria.

The main source of interest, from those who attended, was the development of a medication that targets the disease, stops the production of oxalate, and can help stop the progression of chronic kidney disease.

Highlights of the article’s interview with Douglas Fambrough, Ph.D., of Dicerna, can be found here. A more in-depth interview can be found here.

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