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- PMLiVE

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- PMLiVE

Roche’s spinal muscular atrophy therapy Evrysdi approved by EC for newborns

The neuromuscular disease affects approximately one in every 10,000 babies

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

- PMLiVE

New NHS treatments increase survival rates for babies with spinal muscular atrophy

Around 70 babies are born with the rare muscle-wasting disease each year in the UK

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

Sandoz granted positive CHMP opinion for multiple sclerosis biosimilar

The neurological disease affects approximately 2.8 million people worldwide

- PMLiVE

Novartis acquires DTx Pharma in deal worth up to $1bn

The agreement gives Novartis access to three preclinical neuroscience programmes

- PMLiVE

LifeArc launches rare disease programme with £100m investment

An initial £40m will go towards the creation of up to five rare disease research centres

- PMLiVE

Horizon shares positive results for Uplizna in neuromyelitis optica spectrum disorder

The rare and debilitating disease affects approximately one to ten per 100,000 people

- PMLiVE

NIHR and MRC establish UK Rare Disease Research Platform with £14m investment

The platform brings together expertise from across the UK rare disease research system

- PMLiVE

Novartis to sell eye-care products to Bausch + Lomb in deal worth up to $2.5bn

The transaction includes the Swiss drugmaker’s anti-inflammatory eye drop Xiidra

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