The quest for solid tumor CAR-T
For all their success in hematological cancers, the track record of CAR-Ts against solid tumors has been disappointing (though see here for an exception). One key obsta…
How the US taxpayer saved 25M+ people
A short piece celebrating the 20th anniversary of PEPFAR, a program that few outside of the field of Global Health are aware of. The world is not always nasty and…
Download a PDF of this article here.
Hematologic cancers account for ~10% of annual new cancer diagnoses and continue to have some of the poorest overall outcomes, particularly for older adults.[1] Ho…
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Ex vivo gene-therapies have been a game-changer for aggressive hematologic cancers. These products can effectively cure patients who otherwise had very low chance …
Gene therapy in beta-thalassemia and sickle cell anemia
Beta-thalassemia and sickle cell anemia are common genetic diseases of hemoglobin (Hb) which manifest themselves in the former through transfusi…
In search of a diagnosis: deploying genomics at scale
A substantial number of children have a disease identified as “rare” without having any kind of causative diagnosis (autism is not a causative dia…
Stunting and the microbiome
Stunting (low growth for age) affects > 20% of children across the globe and has major impact on the brain, on health, and on opportunities for success in life. The prec…
Keeping up with COVID-19
It’s not easy for a refereed weekly print periodical to keep up with an epidemic that evolves on a daily basis, but the NEJM is doing its best and all articles are free on-lin…
Are we nearing an asymptote with implantable cardiac pumps?
Severe heart failure is common, and spare hearts for transplant are rare, which has led to the development of implantable mechanical alterna…
Successes in gene therapy for hemophilia B and A
Hemophilia A and B are X-linked genetic diseases which prevents the formation of functional coagulant factors VII and IX respectively and cause a prope…
Two new therapies against a horrible congenital disease – but trouble ahead on pricing…
Spinal Muscular Atrophy (SMA) is a genetic disease that declares itself at a few months of age, and typically le…
Gene therapy for sickle cell disease
Typical diseases targeted by gene therapy are those for which there is a defect that prevents the production of a functional protein needed for normal life; remedi…
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