Rare Disease

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Centessa plans $100m IPO to fund R&D and potential acquisiti...

Centessa has announced plans for a $100m US IPO just weeks after the company launched with $250m in private backing and an R&D powerhouse driven by the merger of 10 biotechs.

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Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Scotland opens door to Translarna for Duchenne muscular dyst...

A disparity in UK access to a medicine for Duchenne muscular dystrophy (DMD) has been addressed, after Scotland gave a green light to use of PTC Therapeutics' Translarna for the first time.

News

NICE backs Sobi's Kineret for rare inflammatory disease

The UK's cost-effectiveness agency NICE has recommended that Sobi's Kineret can be used to treat NHS patients with Still's disease, a rare form of arthritis affecting children and adults.

Views & Analysis

Solving the “information challenge” of rare disease diagnosi...

Getting a diagnosis is often the biggest challenge facing a rare disease patient in the UK.

News

Protein misfolding specialist Gain nets $40m from IPO

Gain Therapeutics has priced its initial public offering, raising more than $40 million for its computational drug discovery platform and pipeline of drugs for diseases associated with prot

Views & Analysis