Beren Therapeutics P.B.C., through its subsidiary Mandos LLC, has announced that the U.S. Food and Drug Administration (FDA) has officially accepted its New Drug Application (NDA) for adrabetadex. Read More
The scientific community is preparing for the highly anticipated 5th National Symposium on Genetic Diseases (NSGDCON). Save the Date: October 24th & 25th, 2026 Read More
The Division of Genetics, Department of Pediatrics at the All India Institute of Medical Sciences (AIIMS), New Delhi, has announced the launch of a significant interventional clinical trial: ELEGANT. Read More
A new Phase 3 study, titled NAVIGATE, is currently evaluating the efficacy and safety of Nizubaglustat for patients with late-infantile and juvenile forms of Niemann-Pick Type C (NPC) and GM1/GM2 Gangliosidosis. Read More
The National Neonatology Forum (NNF) and leading clinical geneticists have debuted a monthly scientific series. The initiative aims to revolutionize NICU outcomes through expert-led sessions on genomic testing, metabolic emergencies, and dysmorphology. Read More
Kerala becomes the first Indian state to earmark ₹30 crore in its budget for Spinal Muscular Atrophy (SMA) treatment, shifting from crowdfunding to state-backed support for rare diseases. Read More