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Results For "rare-disease"

198 News Found

EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours
Clinical Trials | October 31, 2025

EU approves Alexion’s Koselugo to treat adults with neurofibromatosis type 1 tumours

Approval based on pivotal KOMET Phase III trial demonstrating significant tumour reduction in adults with NF1


Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions
Clinical Trials | October 31, 2025

Alexion highlights breakthrough C5 inhibition advances in gMG at 2025 AANEM and MGFA scientific sessions

New Phase III data on gefurulimab highlight potential as a self-administered treatment option; real-world evidence reinforces clinical benefits of Ultomiris and Soliris


Sanofi's Q3 profit dips marginally as Dupixent sales top €4 billion
News | October 27, 2025

Sanofi's Q3 profit dips marginally as Dupixent sales top €4 billion

The company's net income attributable to equity holders dropped to €2.80 billion from €2.82 billion last year


Novo Nordisk acquires Omeros’ MASP-3 inhibitor Zaltenibart for up to US$ 340 million
News | October 17, 2025

Novo Nordisk acquires Omeros’ MASP-3 inhibitor Zaltenibart for up to US$ 340 million

Zaltenibart offers best-in-class potential across multiple rare blood and kidney disorders, enhancing Novo Nordisk’s rare disease portfolio


Delhi High Court refuses to restrain Natco from manufacturing spinal muscular atrophy drug
News | October 10, 2025

Delhi High Court refuses to restrain Natco from manufacturing spinal muscular atrophy drug

The Court dismissed Swiss drugmaker Roche’s plea for interim injunction


India invites applications under PRIP scheme to drive pharma-medtech innovation
R&D | October 06, 2025

India invites applications under PRIP scheme to drive pharma-medtech innovation

Early stage projects of up to Rs. 9 crore eligible for up to Rs. 5 crore assistance and later stage projects of up to Rs. 285 crore eligible for up to Rs. 100 crore assistance


Moderna opens manufacturing and R&D facility in UK
News | September 27, 2025

Moderna opens manufacturing and R&D facility in UK

The Moderna Innovation and Technology Centre in Harwell, Oxfordshire is now fully operational and Moderna is licensed to supply British-made COVID-19 vaccines to the UK population


Datavant partners with Indegene to power clinical trial recruitment
Digitisation | September 21, 2025

Datavant partners with Indegene to power clinical trial recruitment

Collaboration matches more eligible patients, including those with rare and complex diseases, to appropriate clinical trials


FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia
Drug Approval | September 02, 2025

FDA approves Sanofi’s BTK inhibitor for immune thrombocytopenia

ITP is a disease of complex immune dysregulation leading to low platelet counts, bleeding, and reduced quality of life


Sarepta advances siRNA collaboration and sale of arrowhead equity investment
News | August 19, 2025

Sarepta advances siRNA collaboration and sale of arrowhead equity investment

Sarepta has also entered into an agreement with Arrowhead to transfer 2,660,989 shares of Arrowhead common stock in satisfaction of $50 million of the company’s previously announced $100 million milestone payment obligation