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- PMLiVE

EURORDIS

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

Biomarin

EC approves Biomarin rare genetic disorder drug

Vimizim now available to treat Morquio A syndrome

- PMLiVE

FDA approves Janssen drug for rare Castleman’s disease

Sylvant approved to treat lymphoma-like condition

- PMLiVE

BioAlliance and Topotarget merge to focus on rare cancers

French and Danish firms combine to develop orphan oncology drugs

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