by Peter Ciszewski | Jun 6, 2018
Bernice Morrow, PhD, Director of Translational Genetics, Albert Einstein College of Medicine, discusses some of the challenges of diagnosing patients with 22q11.2 deletion syndrome, a disorder caused by the deletion of a small piece of chromosome 22. 22q11.2 deletion...
by Peter Ciszewski | Jun 5, 2018
Dr. John Maraganore has served as the CEO and a Director of Alnylam since 2002 and is the Chair of the Biotechnology Innovation Organization (BIO) for the 2017-2018 term. Dr. John Maraganore has led a career pursuing therapies to address unmet medical needs. At...
by Peter Ciszewski | Jun 3, 2018
The FDA has granted Breakthrough Therapy designation to Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy (CALD), a rare, serious and life-threatening hereditary neurological disorder. This FDA designation is designed to quicken the development...
by Peter Ciszewski | Jun 2, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is a progressive condition that affects most tissues and organs. The severity...
by Peter Ciszewski | Jun 1, 2018
Daniel de Boer, Founder and CEO of ProQR, provides an overview of his company. ProQR’s focus is on diseases that are very severe or life threatening, and have limited treatment options. The company is developing new therapies for several forms of...
