Rare diseases

Views & Analysis

Millendo’s Julia Owens on leadership in rare diseases

Rare disease specialists Millendo Therapeutics are hoping their drug livoletide will become the first treatment for Prader-Willi syndrome.

News

Chiesi starts rare diseases division, ahead of potential Fab...

Italian pharma Chiesi has begun a new rare diseases division as it builds towards a potential commercial launch for its Fabry Disease therapy.

News

Child with rare disease takes NICE to court over access to l...

NICE’s decision-making process will come under legal scrutiny in court this week, after a child and her family claimed the NHS cost-effectiveness body is unlawfully limiting access to a lif

Events

Partner Content

Real-World Evidence 2020: Rare Diseases and Innovative Thera...

Explore practical applications of RWE in rare diseases

Partner Content

Partner Content

Life with multiple myeloma

Multiple myeloma patient Bob Munro (pictured below) explains what it's like to live with the rare blood cancer and how his diagnosis led him on the journey of a lifetime – cycling from

News

Akcea's rare disease drug Waylivra way too pricey for NHS, s...

Less than a week into 2020 and NICE has provisionally ruled against NHS funding for another drug – in this case Akcea’s ground-breaking antisense drug Waylivra (volanesorsen) for the ultra-

Millendo’s Julia Owens on leadership in rare diseases

Chiesi starts rare diseases division, ahead of potential Fab...

Child with rare disease takes NICE to court over access to l...

Real-World Evidence 2020: Rare Diseases and Innovative Thera...

Life with multiple myeloma

Akcea's rare disease drug Waylivra way too pricey for NHS, s...

Delivering the right behavioural ingredients for the moment,...

How to meet your physicians where they are online

Rare diseases

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