Rare diseases

Market Access

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Achieving launch excellence in orphan medicines

New rare disease treatments face a complex market access landscape that presents fundamental challenges for pharmaceutical companies seeking to achieve launch excellence.

News

Under threat from Novartis, Biogen hails new data from SMA d...

Under threat from a gene therapy from Novartis, Biogen is making the case for its drug Spinraza for the muscle wasting disease spinal muscular atrophy, with new data that it hopes persuades

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Alexion's Soliris gets new, and lucrative, rare disease indi...

Alexion’s rare disease drug Soliris has a new indication after the FDA approved it for a disease that attacks the optic nerves.

Partner Content

Partner Content

Alexion Pharmaceuticals & Minoryx Therapeutics to Present at...

SMi Reports: Alexion Pharmaceuticals and Minoryx Therapeutics to deliver presentations at the 9th Annual Orphan Drugs and Rare Diseases Conference

News

Spain's Sanifit raises €72 million for rare disease drug tri...

Sanifit has raised €72.2 million ($80.9m) in Spain’s largest private biotech fundraiser to finance phase 3 clinical development of its rare disease drug SNF472.

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