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- PMLiVE

Wakix

- PMLiVE

Merck to acquire Caraway Therapeutics in a deal worth up to $610m

The deal gives Merck access to a pipeline of therapeutics for neurodegenerative and rare diseases

- PMLiVE

NICE recommends Chiesi’s alpha-mannosidosis enzyme replacement therapy

Alpha-mannosidosis, an extremely rare inherited condition characterised by a broad range of impairments, affects approximately 25 people in England

- PMLiVE

Infographic: Rare Diseases in China

How rare are rare diseases in China? Download this infographic to find out more and how we can offer guidance for pharma companies to consider.

Research Partnership

- PMLiVE

Landscape review reveals £1.1bn of UK funding for rare diseases

The report focuses on future funding and needs for those with rare diseases

- PMLiVE

Clinigen launches educational programme for rare disease patients and organisations

The NaviGATE programme will aim to empower patients and provide navigational support

- PMLiVE

Pfizer and BioNTech’s XBB.1.5-adapted COVID-19 vaccine recommended by CHMP

Doses will be ready to ship immediately upon authorisation by the European Commission

regeneron headquarters

Regeneron’s Veopoz granted FDA approval for ultra-rare immune disease

Fewer than ten patients in the US have been diagnosed with CHAPLE disease

Biogen Idec building

Biogen to acquire Reata for approximately $7.3bn

The deal gives Biogen access to a recently launched therapy for Friedreich's ataxia

- PMLiVE

AstraZeneca’s rare disease unit to acquire Pfizer’s early-stage gene therapies for $1bn

The transaction could potentially see staff associated with the portfolio move to Alexion

- PMLiVE

Roche’s spinal muscular atrophy therapy recommended by CHMP for newborns

Evrysdi is already approved in the EU to treat SMA patients aged two months or older

- PMLiVE

Bristol Myers Squibb’s Opdivo recommended by CHMP for expanded melanoma use

The global incidence of skin cancer has been rising steadily over the past few decades

- PMLiVE

Merck granted positive CHMP opinion for Keytruda combination in gastric cancer

Over one million new cases of gastric cancer were diagnosed globally in 2020

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