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CTRS

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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

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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

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

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AstraZeneca announces major expansion of Canadian research footprint

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

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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

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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

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

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The Need for and Impact of Rare Disease Education

Physicians are often told that when they hear hoofbeats, think horses, not zebras — to look for the most common cause of a patient’s symptoms first. While this advice may...

Medscape Education

Medscape Education: Rare Diseases Fibrodysplasia Ossificans Progressiva

Rare Disease Day is on February 28.Listen to Nicky explain her daughter's journey with fibrodysplasia ossificans progressiva.Many patients face long waits for accurate diagnosis and treatment, making it critically important...

Medscape Education

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SMC enables access to Kyowa Kirin’s Crysvita for adults with rare genetic disease

XLH is a life-long genetic disease that causes abnormalities in the bones, muscles and joints

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