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Day-1
Sessions
Treatment and Advanced Therapies for Rare Diseases | Economic aspects of Rare Diseases and Orphan
Drugs | Rare Nutritional and Metabolic Diseases | Rare Musculoskeletal Diseases | Rare Hereditary Diseases
Chair:
Vesna Aleksovska,
International Gaucher Alliance, Macedonia
Session Introduction
Title:
Red blood cell-encapsulated enzymes: An innovative therapeutic approach to overcome
challenges of enzyme replacement therapies for rare diseases
Emmanuelle Cecile Dufour,
Erytech Pharma, USA
Title:
The Economics and Sustainability of Orphan Drugs
Carina Schulmann Schey,
University of Groningen, Netherlands
Title:
New therapies in genetic skeletal diseases achieved through drug repurposing
Michael Darren Briggs,
Newcastle University, UK
Title:
Nitisinone in the treatment of alkaptonuria
Lakshminarayan Ranganath,
Royal Liverpool University Hospital, UK
Title:
Change in gonadotropins in postmenopausal women: Effects of parity
Ekhator C N,
Ambrose Alli University, Nigeria
Title:
Clinical case of congenital hyperinsulinism in infant born by mother with type 2 diabetes
N B Belykh,
Omsk State Medical University, Russian Federation