by Victoria Radke | Jan 22, 2021
PTC Therapeutics recently opened applications for the seventh annual STRIVE Awards program for Duchenne muscular dystrophy. Duchenne muscular dystrophy is the most common and most severe form of muscular dystrophy, caused mutations in the DMD gene that leads to loss...
by Victoria Radke | Jan 21, 2021
The U.S. Food and Drug Administration (FDA) has approved Janssen’s daratumumab and hyaluronidase-fihj combination therapy for adults with newly diagnosed amyloid light chain (AL) amyloidosis. AL amyloidosis is a rare blood disorder associated with the overproduction...
by Victoria Radke | Nov 30, 2020
The U.S. Food and Drug Administration (FDA) has approved Rhythm Pharmaceuticals’ setmelanotide for chronic weight management in patients 6 years and older with obesity due to the following three rare conditions: pro-opiomelanocortin (POMC) deficiency, proprotein...
by Victoria Radke | Nov 26, 2020
The U.S. Food and Drug Administration (FDA) has approved naxitamab (Danyelza) to treat neuroblastoma in the bone or bone marrow. Naxitamab, a monoclonal antibody that targets the ganglioside GD2, is approved in combination with granulocyte-macrophage...
by Victoria Radke | Nov 13, 2020
Molly and Adam Fleming, the parents of Natalie, a young girl with Rett syndrome, discuss Natalie’s diagnostic journey. Rett syndrome is a rare progressive neurodevelopmental condition that primarily affects girls. These girls appear to have normal pyschomotor...
