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Inozyme is a leader in developing therapies for rare diseases.


Understanding the biology of Calciphylaxis is key to changing the treatment paradigm for patients.

Calciphylaxis is a rare disease with a high mortality rate that mostly affects patients with end-stage kidney disease (ESKD). It is caused by medial calcification, intimal proliferation, and thrombosis and is associated with low levels of pyrophosphate (PPi). The disease is characterized by pathologic mineralization (i.e. calcification) and intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels) of the vasculature in the skin and fatty tissue leading to poor blood flow, blood clots, painful skin ulcers, serious infections, and death. Initial skin lesions typically present as extremely painful plaques and nodules, and progress to necrotic ulcers. 

Approximately 50% of Calciphylaxis patients die within a year of diagnosis. More than 70% of individuals require hospitalization for severe ulcerations. An estimated 50% of patients are bedridden or wheelchair-bound.

There are no approved therapies for Calciphylaxis.


Estimated Incidence

Disease Prevalence

3.5: 1,000 dialysis patients

~ 5,000 in Major Markets

Complications Caused by Calciphylaxis

Poor Blood Flow

Calcium deposits form in blood vessels and block blood flow, leading to areas where skin and tissue break down and die.

Blood Clots

Recent studies show that most people with Calciphylaxis have abnormalities in blood-clotting factors. These abnormalities can lead to small blood clots forming more often than they normally would. Calcium is deposited in the smallest parts of the arteries, which also leads to the formation of blood clots.

Skin Ulcers

Deep, painful lumps ulcerate and create open sores that fail to heal. These ulcers often occur on the stomach and thighs but can appear anywhere on the body.

Serious Infections

Serious infections occur due to wounds on the skin that spread and do not heal, which can lead to major complications including sepsis, the most common cause of death.