Rare diseases

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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

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

Explore practical applications of RWE in rare diseases

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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-

News

Alnylam to file rare kidney disease drug after trial win

After years where RNA interference research was in the doldrums, Alnylam is on a roll and is preparing to file a rare kidney disease drug with the FDA, after two previous approvals.

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...

Alnylam to file rare kidney disease drug after trial win

Biotech is back, with Craig Ackerman

Using AI-driven synthetic personas to take your insights fur...

Rare diseases

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