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- PMLiVE

Lamzede

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EMA offers orphan drug benefits for Ebola research

Incentives to encourage development of new treatments for the virus

- PMLiVE

FDA awards $19m in rare disease grants

US regulator supports 15 orphan drug projects

- PMLiVE

NICE backs Celgene’s Revlimid in bone marrow disorder

Recommends that drug can be used on NHS to treat myelodysplastic syndromes

Genentech’s Sunil Agarwal joins rare disease firm

He will serve as chief medical officer at Ultragenyx

- PMLiVE

FDA approves drug for rare swelling diseases

Salix and Pharming’s Ruconest is made from the milk of genetically-modified rabbits

- PMLiVE

Channel strategy and orphan drugs

A plan for guiding decisions about a product's distribution channel is particularly relevant for orphan drugs, rare diseases or specialty medicines

- PMLiVE

FDA sets out framework for rare childhood diseases

Wants to encourage more trial use of biomarkers and speedier approval timelines

- PMLiVE

The moving parts of orphan drug development

Tackling rare diseases requires high levels of cooperation, regulatory support and a determination to succeed

- PMLiVE

FDA gives early green light to Spectrum’s lymphoma drug

Beleodaq approved to treat rare cancer

- PMLiVE

Otsuka launches European ADPKD forum

Aims to lead awareness raising plans for rare genetic condition

- PMLiVE

EC approves Janssen rare blood disorder drug

Sylvant available to treat multicentric Castleman's disease

Shire Basingstoke

Shire boosts rare disease portfolio with $260m Lumena deal

Acquisition of US company will bring two mid-stage candidates for rare liver diseases

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