Pipeline options aplenty for people with Fabry disease
Updated results from the Phase I/II STAAR trial are good enough for Sangamo Therapeutics (Nasdaq: SGMO) to trigger the expansion phase of the study.
The Californian genetic medicine company is also planning for a possible late-stage study for its candidate, ST-920, a wholly-owned gene therapy targeting Fabry disease.
People with the rare lysosomal storage disorder have abnormal enzyme activity, leading to episodes of pain and a range of potentially life-threatening complications, such as progressive kidney failure, heart failure, and stroke.
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