by Peter Ciszewski | Jan 21, 2019
Charcot-Marie-Tooth disease (CMT) is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the United States. The disease is named for the three physicians who first identified it in 1886 – Jean-Martin...
by Peter Ciszewski | Jan 16, 2019
Despite a government shutdown, the FDA approved cabozantinib (Cabometyx) as a treatment for patients with hepatocellular carcinoma (HCC) who previously received sorafenib (Nexavar). The approval was based on findings from the phase III CELESTIAL trial, in which...
by Peter Ciszewski | Jan 16, 2019
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City provides an overview of Mucopolysaccharidosis type IV and VI (MPS IV and VI). Mucopolysaccharidosis type IV is also known as Morquio syndrome, is a progressive...
by Peter Ciszewski | Jan 15, 2019
The U.S. Food and Drug Administration (FDA) has granted fast track status to investigational therapy CTX001 to treat sickle cell disease. This Phase 1/2 clinical trial (NCT03745287) is currently recruiting patients. CTX001 uses the CRISPR gene-editing technology to...
by Peter Ciszewski | Jan 11, 2019
In the first study of its kind, researchers explored patients’, parents’ and physicians’ perspectives on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease (SCD). CRISPR-Cas9 is a gene editing technology that researchers have...
