by Peter Ciszewski | Jun 28, 2017
Sharon Terry is President and CEO of Genetic Alliance, a network of more than 10,000 organizations, and the founding CEO of PXE International, a research advocacy organization for the genetic condition pseudoxanthoma elasticum (PXE), which affects Terry’s two adult...
by Peter Ciszewski | Jun 28, 2017
Amicus Therapeutics has submitted a Japanese new drug application (J-NDA) to the Pharmaceuticals and Medical Devices Agency (PMDA) to request marketing authorization for migalastat, an oral precision medicine for Fabry disease. John F. Crowley, Chairman and Chief...
by Peter Ciszewski | Jun 28, 2017
There may be new hope on the horizon for those suffering from Huntington’s Disease. Recent research at Emory University, using a gene editing tool called CRISPR-Cas9, has provided new insight into how the disease progresses, and possible ways to reverse its...
by Peter Ciszewski | Jun 25, 2017
Pfizer’s generic injectables company, Hospira, received a complete response letter (CRL) for their proposed biosimilar of epoetin alfa, also known as Epogen and Procrit, following manufacturing issues cited by the Food and Drug Administration (FDA) in a warning...
by Peter Ciszewski | Jun 23, 2017
The U.S. Food and Drug Administration today approved Haegarda, the first C1 Esterase Inhibitor (Human) for subcutaneous administration to prevent Hereditary Angioedema (HAE) attacks in adolescent and adult patients. The subcutaneous route of administration allows for...
