Ionis gets approval for preventing attacks in rare and genetic HAE
In Ionis Pharmaceuticals Inc.’s second U.S. FDA approval in under a year, the agency approved Dawnzera (donidalorsen) as…
China Unveils Genome Tools for High-Precision DNA Editing in Humans, Crops
(Yicai) Aug. 7 — Chinese researchers have developed two advanced DNA editing tools that could lead to breakthroughs…
Novel Human Genetics Evidence Confirms Estimates of Genetic Prevalence, Underdiagnosis, and Potentially Greater Symptom Burden of Gain-of-Function CASR Variants Associated with ADH1
BridgeBio Pharma, Inc. – By examining over 100 unique variants associated with the CASR gene causative of ADH1,…
How India can weaponise data in battle against rare, genetic diseases
India has no prevalence data for rare diseases and very little genetic references for its own population. Arjun…
New test speeds up diagnosis of rare genetic diseases in children
In a major leap for genetic diagnostics, scientists from the University of Melbourne and the Murdoch Children’s Research…
The Role of CRISPR-based Gene Editing
There are an estimated 6,000 Mendelian diseases, most of which still lack cures. Gene editing is an essential…
The Role of CRISPR-based Gene Editing
There are an estimated 6,000 Mendelian diseases, most of which still lack cures. Gene editing is an essential…