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Gaucher Disease Suppliers In Europe
2 companies found
based inBethesda, MARYLAND (USA)
Gain Therapeutics is redefining drug discovery with its SEE-Txâ„¢ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for ...
Gaucher disease is the most common lysosomal storage disease and is caused by mutations in GBA, the gene that encodes the beta-glucocerebrosidase (GCase) enzyme. These mutations result in the ...
based inNaarden, NETHERLANDS
Azafaros has leveraged on decades of discovery by professors Hans Aerts, Hermen Overkleeft, and Stan van Boeckel at Leiden University and the Academic Medical Center in the Netherlands. Expanding on their research and led by highly experienced ...
Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These disorders are individually rare but collectively affect 1 in 5,000 ...