Rare Disease Equipment Supplied In Spain
2 equipment items found
by:Gain Therapeutics, Inc. based inBethesda, MARYLAND (USA)
Mucopolysaccharidosis type 1 (MPS 1) is a rare lysosomal storage disease that is caused by mutations in IDUA, the gene that encodes the alpha-L-iduronidase (IDUA) enzyme. These mutations cause the misfolding and dysfunction of IDUA, which leads to the toxic buildup of large sugars in the bone, cartilage, cornea, heart and central nervous system (CNS). ...
Manufactured by:Suvoda based inConshohocken, PENNSYLVANIA (USA)
Our advanced patient randomization and trial supply management system. Your clinical trial command and control ...