After Elevidys: DMD Advocacy Past First Gene Therapy Approval
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.
Running With Scissors: The FDA Moves to Tame Gene Therapy Drug Development
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.
FDA Approves First Gene Therapy for Hemophilia B
The BLA was supported by positive data from 54 patients with hemophilia B in the pivotal phase 3 HOPE-B trial.
Beti-Cel Gene Therapy for β-Thalassemia Receives FDA Approval
“The approval of ZYNTEGLO marks a watershed moment for the field of gene therapy," Andrew Obenshain, CEO of Bluebird Bio says.
Team Approach Needed for Advancing Treatments for Rare Diseases
Clinical and industry leaders share their perspectives on the importance of collaboration in developing treatments for rare diseases.
A Wrap-Up of Ophthalmologic Cell And Gene Therapies Currently in Development
A multitude of promising new investigative therapies are on the horizon for inherited retinal disease.
The State of Gene and Cell Therapy for Sickle Cell 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.
Pimavanserin Doesn’t Cause Negative Cognition Treating Dementia-Related Psychosis
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 Clarify Alzheimer Disease Biomarkers and MRI Measures
Researchers examine how biomarkers are differentially mapped to MRI measures of cortical thickness.
Sleep Apnea Linked to Visceral Adiposity in Geriatric Patients
Increasing OSA severity was associated with increasing markers of obesity.
Alector Moving Forward with AL002 Alzheimer Disease Trial
Alector’s announcement about the INVOKE-2 study follows a similar update regarding its INFRONT-3 study for frontotemporal dementia.
Various Factors Linked to Increased Idiopathic Intracranial Hypertension Risk
Obese individuals were found to have a higher odds ratio of developing IIH compared to non-obese people.
Small Vessel Disease Increases Risk of Ischemic Stroke
Patients with any presence of small vessel disease had a 2-times greater risk of recurrent ischemic stroke than those without.
Particulate Matter Increases Future Risk of Alzheimer Disease
Researchers from USC propose a direct neurotoxic effect of particulate matter smaller than 2.5 μm.
2 Clarke Drive Cranbury, NJ 08512