- PMLiVE

Chiesi launches new rare disease R&D division

New business unit will be based in Boston, US

Sanofi reception

Sanofi’s rare disease drug olipudase alfa scores in late-stage study

Met crucial endpoints across two clinical studies

- PMLiVE

Abeona Therapeutics’ rare disease phase 3 trial halted due to FDA concerns

Issues over transport stability must be resolved, says regulator

- PMLiVE

bluebird bio reveals further encouraging data for CALD gene therapy

Study shows continued benefit in treatment of rare disease

- PMLiVE

Pfizer goes big on gene therapy with $500m facility spend

Continues to build on its growing gene therapy business

- PMLiVE

Another lost decade for research?

What does the patent landscape look like for antibiotics, vaccines and orphan drugs?

- PMLiVE

First von Willebrand disease therapy heads new FDA approvals

Baxalta's Vonvendi set for an early 2016 launch in the US

EU flag

Wakix backed for European approval in narcolepsy

Bioprojet Pharma’s orphan drug given a positive opinion by CHMP

- PMLiVE

Orphan drug firm Sobi opens new European base

Company'sBrussels office will also house its Benelux operations

- PMLiVE

FDA grants Tocagen’s glioblastoma drug orphan status

Puts the brain cancer gene therapy in-line for development incentives

- PMLiVE

Sobi wins European approval for oral form of orphan drug Orfadin

Expandstreatment options for rare genetic disease HT-1

- PMLiVE

Ten new medicines recommended in Europe

Includes double approvals for rare disease specialist Alexion and Novartis

- PMLiVE

Alexion expands in Ireland with new biofacility

New manufacturing facility should be ready in the next four years

- PMLiVE

Alexion boosts rare disease portfolio with $8.4bn Synageva buy

Deal will help bolster firm's offering which is currently headed by aHUS drug Soliris