Jesse Shefferman

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rare diseases parenteral nutrition choline deficiency metabolic diseases orphan diseases gastrointestinal diseases short bowel syndrome drug development corporate development nonmuscle invasive bladder cancer lymphatic malformations biotechnology: biological products (no diagnostic substances) health care biotechnology research cell therapy non-muscle invasive bladder cancer nmibc lms iv choline chloride fda approval clinical trials investigational therapy parenteral support orphan drug designation high-grade carcinoma immunopotentiator tumor necrosis factor pro-inflammatory response cytokines safety evaluation efficacy studies biopharmaceutical intravesical instillation phospholipid substrate replacement hepatology gene expression modulation clinical development pediatric patients mixed cystic lms chronic liver disease macrocystic diseases tumor response anti-tumor activity substance approval biologic manufacturing patient access program expanded access transformative therapies health care innovation therapeutic development commitment to patients patient enrollment high unmet need medical research regulatory strategy treatment efficacy clinical data analysis healthcare professionals careers in biotech investor relations health wellness & fitness hospital & health care