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Gene Therapy Products Equipment Supplied In Usa New York
4 equipment items found
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
FA is a rare, genetic disorder affecting DNA repair. Approximately two-thirds of FA cases are caused by genetic defects in the FANCA gene, which results in the FA subtype known as FA Complementation Group A (FA-A). FA patients may develop bone marrow failure (very low blood counts), cancers of the blood or other ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
PKD is a rare, genetic blood disorder. PKD is caused by a defect in the PKLR gene which is responsible for energy production in red blood cells (RBCs). RBCs carry oxygen to the rest of the body. When the PKLR gene has a defect, RBCs are limited in their ability to produce energy and remain intact. As a result, patients with PKD frequently have anemia, chronic fatigue, yellowing of the skin and ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
LAD-I is a rare genetic disorder affecting the immune system and is caused by a defect in the ITGB2 gene, leading to a deficiency in CD18. Due to this deficiency, white blood cells (primarily neutrophils) are unable to leave the bloodstream to go to the site of infection. As a result, patients with severe LAD-I can develop life-threatening infections which they are unable to fight. Without a ...
Manufactured by:Rocket Pharmaceuticals, Inc. based inCranbury, NEW JERSEY (USA)
Danon Disease (DD) is a rare genetic disorder that is characterized by severe and primarily hypertrophic cardiomyopathy. Skeletal muscle weakness and mild cognitive impairment are also common. The causative mutation has been identified in the gene encoding for lysosome-associated membrane protein (LAMP2), and in particular the LAMP2B version of the gene which is primarily expressed in heart, ...