Pharvaris Announces Positive Phase 3 Results for Deucrictibant as On Demand HAE Treatment Pharvaris has announced positive topline data from its global Phase 3 RAPIDe 3 study, confirming that the oral bradykinin B2 receptor antagonist deucrictibant shows strong...
Otsuka Gains Approval for First of Its Kind Treatment Against Rare Kidney Disease In a significant milestone for kidney disease care, Otsuka Pharmaceutical has received accelerated approval from the US Food and Drug Administration for a new therapy for Immunoglobulin...
Engasertib Trial Offers New Hope for Patients with Hereditary Hemorrhagic Telangiectasia A recent clinical study has delivered encouraging results for people living with hereditary hemorrhagic telangiectasia, a rare genetic vascular disorder that causes chronic and...
Pharmaceutical approaches to ALS remain sparse and "hard-to-develop". Riluzole (approved 1995) and edaravone (IV in 2017, oral in 2022) only offer modest benefits. More recently, Biogen's Qalsody (tofersen) was approved for SOD1-mutant ALS. But a new wave of new...
Miraculous Progress for 3-Year-Old After Ground-Breaking Gene Therapy for Hunter Syndrome A three-year-old boy, Oliver Chu, has astounded doctors after becoming the first person in the world with Hunter syndrome also called MPS II to receive a pioneering gene therapy....
Clinical trials have long been the backbone of medical innovation, providing the evidence base for new treatments, diagnostics, and preventive measures. Yet for decades, the traditional model, site-based, paper-driven, and geographically limited, has been plagued by...
Clinical trials have long been the backbone of medical innovation, providing the evidence base for new treatments, diagnostics, and preventive measures. Yet for decades, the traditional model, site-based, paper-driven, and geographically limited, has been plagued by...
In a ground breaking advance in reproductive biology, researchers in the United States have for the first time created early-stage human embryos by converting DNA from skin cells into functional eggs and then fertilising them with sperm. Though still at an...
In a pioneering medical achievement, a 25-year-old woman in China has become the first person in the world to reverse Type 1 diabetes using stem cells derived from her own body. The groundbreaking study, published in Cell in September 2024, marks a turning point in...
In a historic stride toward personalised cancer treatment, BioNTech—the German biotech giant behind one of the first COVID-19 mRNA vaccines—has launched the world’s first mRNA-based lung cancer vaccine, BNT116, into Phase I clinical trials across seven countries. This...