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- PMLiVE

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EMA recommends revoking marketing authorisation for Novartis’ sickle cell disease drug

The EU regulator concluded that the benefits of Adakveo did not outweigh its risks

- 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

Novartis’ Cosentyx recommended by CHMP for hidradenitis suppurativa

An estimated one in 100 people globally are affected by the inflammatory skin disease

- PMLiVE

Novartis announces positive long-term data for relapsing MS treatment Kesimpta

Approximately 85% of MS patients are initially diagnosed with relapsing forms 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

- PMLiVE

AstraZeneca announces major expansion of Canadian research footprint

The investment includes the creation of 500 jobs and a new rare disease research hub

- PMLiVE

Rare Disease Day 2023 – raising awareness around the world

Around one in 17 people will be affected by a rare disease at some point in their lives

- PMLiVE

UK government announces new rare diseases action plan for England

The framework lists 13 actions that have been developed with the rare disease community

Medscape Education: Rare Diseases PAH

Today is Rare Disease Day, February 28.Hear from Helen as she describes in her own words what it’s like to live with PAH, and her perspective as a patient.  To learn...

Medscape Education Global

Medscape Education and M4RD: Together Caring for Rare Disease

We are excited to finally share our news-style video ' ', developed in partnership with Medics 4 Rare Diseases (M4RD) and produced by Genetic Alliance UK and ITN Business.For more information on...

Medscape Education Global

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