Barry Greene, President of Alnylam, discusses his company and it’s pending approval of Patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of hereditary ATTR amyloidosis (hATTR amyloidosis).
Luca Santarelli, MD of Therachon discusses achondroplasia, an autosomal dominant disorder characterized by dwarfism and disproportionate shortness of limbs, accompanied by life-altering complications.
John Schall is Chief Executive Officer of Caregiver Action Network (CAN) and in this video he discusses the mission of the CAN and the many valuable resources his group offers. The mission of the Caregiver Action Network is to promote resourcefulness and respect for the more than 90 million family …
Daniel de Boer, Founder and CEO of ProQR, discusses his company’s focus on Leber congenital amaurosis. This rare eye disorder primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color.
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of his company’s leading drug candidate, ELX-02 for cystic fibrosis.