Rare diseases
Alnylam gives up on Onpattroās US expansion after FDA rejects its rare heart disease indication
Despite receiving an endorsement from an advisory committee, the FDA has reached a different conclusion regarding the benefit-risk profile of ...
Amgen Successfully Completes $27.8B Acquisition of Horizon After FTC Dispute Resolution
Amgen has successfully concluded its monumental $27.8 billion acquisition of Horizon Therapeutics, triumphing over a legal challenge posed by the ...
Novartisā iptacopan impresses in phase 3 trial for rare kidney disease
Novartis is celebrating a significant achievement as its investigational oral factor B inhibitor reaches a crucial milestone in a phase ...
Ionisā olezarsen slashes triglycerides and pancreatitis in rare disease patients
Ionis Pharmaceuticals has achieved a significant milestone in its phase 3 clinical trial for lipid-lowering therapy, bringing the company closer ...
Filspari fails to confirm kidney benefit in IgAN, but Travere hopes to keep FDA approval
Half a year after securing accelerated approval for the treatment of a rare kidney ailment, Travere Therapeuticsā groundbreaking drug, Filspari, ...
Clinigen launches initiative to bridge the gap between rare disease communities and pharma
Clinigen, a pharmaceutical services company based in the UK, is launching the NaviGATE initiative to empower patient advocacy groups within ...
AstraZeneca and Verge partner to discover AI-driven drugs for rare neurodegenerative diseases
Verge Genomics is expanding its roster of Big Pharma partnerships by teaming up with AstraZeneca in a substantial $42 million ...
FDA Grants Approval to Ipsen’s Sohonos Capsules, the First-Ever Treatment for Individuals with Fibrodysplasia Ossificans Progressiva
In 2019, Ipsen made a significant investment of $1 billion to acquire Clementia Pharmaceuticals and its rare disease drug, palovarotene. ...
Soft Tissue Sarcoma: Symptoms, Types, Causes, Treatment and Future Directions
What is Soft Tissue Sarcoma? Vascular tumors are a type of soft tissue sarcoma that can affect the blood vessels ...
Vaginal Cancer: A Comprehensive Guide to Symptoms, Types, Causes, Treatment and More
Vaginal cancer is a rare but serious condition that affects the muscular tube that connects your uterus with your outer ...
