This is the question I–along with co-authors Syvart Dennen, Priti Pednekar , Kelly Birch, Menaka Bhor, Julie Kanter , and Peter Neumann–aim to answer in our new paper in JMCP. The full title is: “For which diseases do broader value elements matter most? An evaluation… Read More »For which diseases do broader value elements matter most?
sickle cell disease
I provide the answer to this question in a recent paper in Current Medical Research & Opinion with co-authors Howard Thom, Edna Keeney, Daisy Gaunt, Lauren Zhao, Menaka Bhor, Avery Rizio, Lanetta Bronté-Hall and Nirmish Shah. The abstract is below. AimSickle cell disease (SCD) is… Read More »How do pain crises affect quality of life and productivity among patients with sickle cell disease?
In acquiring the two early-stage Sanofi drugs for sickle cell disease, Global Blood Therapeutics aims to complement Oxbryta, its FDA-approved treatment for the rare blood disorder. Meanwhile, Sanofi can turn its full sickle cell efforts on a gene-editing therapy partnered with Sangamo Therapeutics.
Novartis Collaborates with Bill & Melinda Gates Foundation to Discover and Develop Gene Therapy for Sickle Cell Disease
Shots: The Gates Foundation will provide funding support to discover and develop in vivo gene therapy for SCD. The alliance brings together Novartis drug discovery and gene therapy expertise with Gates’ funding’s The focus of the agreement to address the disparity in access to treatments… Read More »Novartis Collaborates with Bill & Melinda Gates Foundation to Discover and Develop Gene Therapy for Sickle Cell Disease
Cancer is a known risk of lentiviral-based gene therapies. Though bluebird says its gene therapy is engineered to avoid that risk, the company is suspending two clinical trials pending the outcome of its inquiry.
A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting. The data with the CRISPR/Cas9 drug CTX001 is… Read More »ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders
2020 has been an exciting year for gene therapies targeting blood disorders. Despite FDA’s recent rejection of BioMarin’s hemophilia gene therapy program, the clinical landscape is progressing quickly with Pfizer and Sangamo kicking off their phase 3 and bluebird bio receiving European approval for their… Read More »2nd Gene Therapy for Blood Disorders
The post Navigating systemic racism in Canadian healthcare appeared first on Healthy Debate.