The reality is that with nearly 11,000 identified rare diseases, with many that have only dozens of diagnosed patients, the economics of drug discovery and development simply don’t work within existing business models. But don’t tell that to the parents and family members of kids born with a rare disease.…

Life science research changes frequently, with new discoveries being published every day. Some of these discoveries form the basis of new biotech companies, but whether or not these companies will be successful can be hard to predict. Isabelle de Cremoux, CEO of French VC investment firm Seventure Partners, knows this…

A treatment for Fabry disease, a rare X-linked genetic disease that causes progressive buildup of a fatty substance called globotriaosylceramide around the body, has achieved good results at Phase III, report developers Protalix BioTherapeutics and Chiesi Global Rare Diseases. Fabry disease occurs in one person per 40,000-60,000 people and is…

Life science research changes frequently, with new discoveries being published every day. Some of these discoveries form the basis of new biotech companies, but whether or not these companies will be successful can be hard to predict. Isabelle de Cremoux, CEO of French VC investment firm Seventure Partners, knows this…

Researchers based at Wuhan University in China have discovered a mechanism that drives fatty liver disease. They also presented evidence about a small molecule that can target this mechanism, blocking the disorder’s progression in small and large animals. The team’s results suggest this may point to a promising new therapeutic…

A striking feature of the SARS-CoV-2 virus that has caused so much havoc around the world over the last year is how variable a response it elicits from those it infects, with some not even expressing symptoms and others becoming seriously ill. Could variation in our genetics be the answer…

Research continues to find that the effects of COVID-19 may linger long after hospital discharge. In the latest study, researchers from in England found increased rates of organ damage, or multiorgan dysfunction, in a study of patients discharged from NHS hospitals after COVID-19. Published in The BMJ, the risk for…

Twenty years after the publication of the first draft of the human genome, our views have changed on the cipher for humanity, the identity of our ancestors, the differences among us, and the genomic nuances that can govern disease. Much has been deciphered and plenty remains. It is an opportune…

SARS-CoV-2 infection induces functional changes in platelets that make them more likely to aggregate and cause clots, according to a recent study by researchers at the University of Utah. Those changes may explain some the disease’s most dangerous complications, such as heart attacks and strokes. Until now, little was known…

Scientists at the UNC School of Medicine seeking to understand which cells in the respiratory tract are infected SARS-VoC-2, and how it gets into the lungs in the patients who develop pneumonia, has allowed them to characterize some of the ways it infects the nasal cavity and infects and replicates…

Memory—a key feature of the adaptive immune system—is evident in the innate immune system, too. But it has been unclear whether memory in the innate immune system is specific to previously encountered antigens. Now it appears that antigen-specific memory is in fact an attribute of the innate immune system’s monocytes…

While Previous reports of which medications may influence COVID-19 symptoms and outcomes of patients have been reported on a case-by-case basis, a recent study of more than 1,000 COVID-19 positive patients from China suggests that ACE inhibitors and angiotensin receptor blockers may actually increase the risk of severe COVID-19 complications.…

For patients admitted to the hospital and confirmed to have the new coronavirus disease (COVID-19), the key risk factors for death are: Advanced age Signs of sepsis Blood clotting issues These risk factors come from a new retrospective study that evaluated data records for 191 patients—137 survivors and 54 non-survivors.…

A new study led by researchers at the National Institutes of Health (NIH) adds more weight to previous findings implicating variants of the gene ARMC5 in the increased risk of hypertension in blacks. Researchers identified 17 variants in the gene that were associated with high blood pressure in individuals of…

SAN FRANCISCO—Regeneron Pharmaceticals said the $50 million-plus consortium it has launched with five biopharmas will reshape drug development and even human health beyond simply speeding up exome sequencing of all 500,000 people within the UK Biobank by three years. AbbVie, Alnylam Pharmaceuticals, AstraZeneca, Biogen and Pfizer have agreed to invest…