Gaurav Shah, MD, Chief Executive Officer & President of Rocket Pharmaceuticals explains the advantages that smaller biotech companies have in developing treatments for rare diseases.

Rocket Pharmaceuticals is currently developing gene therapies for a number of rare conditions, including Fanconi  anemia, Danon disease, leukocyte adhesion deficiency-I, pyruvate kinase deficiency, and infantile malignant osteopetrosis.

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