Solid Biosciences Focus on Duchenne Muscular Dystrophy

  Ilan Ganot, Co-Founder, President and CEO of Solid Biosciences provides an overview of his company and it’s focus on Duchenne muscular dystrophy (DMD). Mr. Ganot started Solid in 2013 to find treatments, and potentially a cure, for DMD, a disease that...

Mucopolysaccharidosis Type II (MPS II), Hunter Syndrome

  Heather A. Lau, MD, Assistant Professor, Department of Neurology; Associate Director, Division of Neurogenetics; Director, Lysosomal Storage Disease Program at NYU Langone Health provides an overview of Mucopolysaccharidosis type II (MPS II), also known as...

Dup15q Alliance and Syndrome

  Vanessa Vogel-Farley, Executive Director of Dup15q Alliance discusses her organization, which provides family support and promotes awareness, research and targeted treatments for chromosome 15q11.2-13.1 duplication syndrome. Chromosome 15q11.2-13.1 duplication...