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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 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

- PMLiVE

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 Global

- PMLiVE

Sanofi reports positive new data for multiple sclerosis treatment tolebrutinib

It is estimated that there are over 130,000 people living with the condition in the UK alone

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 Global

- PMLiVE

Sanofi and Sobi’s once-weekly haemophilia A treatment approved by FDA

Current factor VIII therapies usually require patients to receive treatment every two days

- PMLiVE

AstraZeneca/Sanofi’s Beyfortus shows consistent protection against RSV disease in infants

RSV is the most common cause of infant respiratory infections, including pneumonia

- PMLiVE

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

- PMLiVE

Sanofi’s enzyme replacement therapy available on NHS to Pompe disease patients

The rare genetic disorder affects around one in every 40,000 people in the UK

- PMLiVE

Sanofi/Regeneron’s Dupixent approved by EC for eosinophilic oesophagitis

This makes Dupixent the first and only targeted medicine indicated for this condition

- PMLiVE

Sanofi/Regeneron’s Dupixent recommended by CHMP for atopic dermatitis in children

If approved, the medicine would be the first and only targeted option in the EU for this age group

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