Accumulation of fat molecules is detrimental to the cell. Researchers from the Yong Loo Lin School of Medicine, National ...
The protein Spns1 helps transport fat molecules out of lysosomes, preventing harmful buildup and offering insights for rare ...
Scientists from Brigham and Women’s Hospital have now identified a new protein that may contribute to the disease.
The enzyme replacement therapy (ERT) market is projected to grow significantly, rising from US$ 10,707.6 million in ...
AceLink is advancing a small-molecule treatment option for Fabry disease that could provide a more convenient alternative to ...
WashU Medicine researcher Patricia Dickson, MD, studies rare genetic diseases called lysosomal storage disorders. Dickson, ...
LCA-0321 is a LYTAC degrader designed to specifically bind and rapidly deplete anti-TSHR autoantibodies, the underlying cause of Graves’ disease and its extrathyroidal manifestations, including ...
Families of rare disease patients appeal to PM Modi and Health Minister for increased treatment funding support.
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