Victoria Johnson

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

With several newly approved therapies hitting the market and an overflowing pipeline, the FDA has stepped in to help keep the burgeoning world of biotech on track.

The BLA was supported by positive data from 54 patients with hemophilia B in the pivotal phase 3 HOPE-B trial.

“The approval of ZYNTEGLO marks a watershed moment for the field of gene therapy," Andrew Obenshain, CEO of Bluebird Bio says.

Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.

A multitude of promising new investigative therapies are on the horizon for inherited retinal disease.

Exciting new therapies for sickle cell disease are on the horizon. We explored some of the most promising investigational therapies in recognition of National Blood Donor Month.

The study presented at the 2021 AAN Meeting follows the complete response letter issued by the FDA to Acadia Pharmaceuticals due to deficiencies in their sNDA.

Researchers examine how biomarkers are differentially mapped to MRI measures of cortical thickness.

Increasing OSA severity was associated with increasing markers of obesity.

Alector’s announcement about the INVOKE-2 study follows a similar update regarding its INFRONT-3 study for frontotemporal dementia.

Obese individuals were found to have a higher odds ratio of developing IIH compared to non-obese people.

Patients with any presence of small vessel disease had a 2-times greater risk of recurrent ischemic stroke than those without.

Researchers from USC propose a direct neurotoxic effect of particulate matter smaller than 2.5 μm.