Pseudoxanthoma elasticum (PXE) is an autosomal recessive connective tissue disorder caused by pathogenic variants in ATP-binding cassette subfamily C, member 6 (ABCC6). ABCC6 typically exports ATP, ...
LA JOLLA, CALIF. – December 1, 2022 – A Phase 2 clinical trial has started of DS-1211 in individuals with Pseudoxanthoma Elasticum (PXE), a rare multisystem genetic disease that causes calcium ...
A Phase 2 clinical trial has started of DS-1211 in individuals with Pseudoxanthoma Elasticum (PXE), a rare multisystem genetic disease that causes calcium deposits in soft tissue resulting in ...
- Positive interim results in infants and young children with ENPP1 Deficiency showed improvements from baseline in multiple measures of disease, including survival, heart function, and stabilization ...
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