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- PMLiVE

Lamzede

- PMLiVE

Eli Lilly’s Mounjaro receives NICE recommendation to treat type 2 diabetes

Around 180,000 patients with type 2 diabetes could benefit from the new treatment option

- PMLiVE

NICE issues two recommendations for UCB’s inflammatory disease drug

Bimzelx is now recommended to treat certain patients with psoriatic arthritis and axial spondyloarthritis, two forms of chronic inflammatory arthritis

- PMLiVE

NICE recommends Chiesi’s Elfabrio for adults with Fabry disease

Affecting approximately 1,150 people in England, Fabry disease is a rare genetic disease that can lead to progressive damage to vital organs

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

NICE recommends Amryt’s Filsuvez treatment for epidermolysis bullosa

Approximately 670 people in England are expected to benefit from the skin-wound-healing treatment

- PMLiVE

NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy

The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors

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

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

FDA approves UCB’s Rystiggo to treat generalised myasthenia gravis

The rare autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

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