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- PMLiVE

Stem Cell Therapeutics

- PMLiVE

Clinigen launches educational programme for rare disease patients and organisations

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

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

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

- PMLiVE

FDA approves Blueprint Medicines’ Ayvakit for indolent systemic mastocytosis

Ayvakit is now the first and only approved medicine designed to treat the underlying cause of the disease

- PMLiVE

NHS England announces rare genetic disorder fast-tracking service

Patients with inherited white matter disorders will benefit from earlier diagnosis and specialist care

Rare Diseases on Medscape Education: Pustular Psoriasis

Rare Disease Day may be over but our committment to advancing outcomes for patients continues. Watch this video to understand a patient's journey with pustular psoriasis.To learn more about Medscape's rare...

Medscape Education Global

Rare Diseases on Medscape Education: Long-Chain Fatty Acid Oxidation Disorder

Rare Disease Day may be over but our committment to advancing outcomes for patients continues. Listen to a mother explain her daughter's journey with a long-chain fatty acid oxidation disorder.To...

Medscape Education Global

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