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Home » What’s new » Rare disease » Page 9

Rare disease

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News and Trends

Could an Alpaca’s monoclonal antibodies be used to treat an ultra-rare blood disease?

Ablynx's Nanobody platform has used the smaller than human monoclonal antibodies from an alpaca to…

By Dani Bancroft
September 29, 2015 - 3 minutes
News and Trends

UniQure’s cure for rare Lysosomal disease ready for phase III trial

UniQure from the Netherlands has promising results for its enzyme replacement therapy phase Ib/II trial…

By Dani Bancroft
September 22, 2015 - 3 minutes
News and Trends

Alexion launches a duo of new therapies for ultra-rare diseases in the EU

Thanks to Alexion Pharmaceuticals, two ultra-rare diseases finally got the first treatment in the European Union,…

By Philip Hemme
September 2, 2015 - 2 minutes
News and Trends

Newron’s ressurection story continues with a FDA Orphan Drug designation

New success for Newron Pharmaceuticals. The FDA approved Orphan Drug designation to its new chemical…

By Philip Hemme
July 14, 2015 - 2 minutes
News and Trends

Two of Alexion’s rare-disease drugs get the thumbs up from the EU

The European Medicines Agency (EMA) is about to approve a couple of rare diseases drugs…

By Lourdes Riquelme
July 1, 2015 - 2 minutes
News and Trends

Alexion will invest €450 million to expand to Dublin

Alexion Pharmaceuticals will travel across the ocean to build its first ever biologics manufacturing facility outside…

By Lourdes Riquelme
May 12, 2015 - 2 minutes
News and Trends

Wilson Therapeutics on its way to cure Wilson disease

Wilson Therapeutics has closed a €37.4M Series B financing to advance its late-stage development program for…

By Lourdes Riquelme
April 16, 2015 - 2 minutes
News and Trends

AOP Orphan and Confluence Pharmaceuticals, together for Fragile X Syndrome

AOP Orphan Pharmaceuticals and Confluence Pharmaceuticals, signed today a definitive agreement to co-develop and market…

By Lourdes Riquelme
March 23, 2015 - 2 minutes
News and Trends

Baxter’s Bioscience Business Acquires SuppreMol

Baxter has acquired SuppreMol for €200M before working capital and other adjustments. The acquisition includes…

By Lourdes Riquelme
March 5, 2015 - 2 minutes
News and Trends

Highway to Brain

Researchers in Barcelona have developed a shuttle to transport drugs into the brain, running through the…

By Lourdes Riquelme
February 18, 2015 - 2 minutes
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