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Gene Therapy Product Suppliers In Usa New York
1 companies found
based inCranbury, NEW JERSEY (USA)
Advancing an integrated pipeline of genetic therapies that correct the root cause of complex and rare life-threatening childhood disorders. Our mission is to develop first-in-class and best-in-class, curative gene therapies for patients with ...
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 ...