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- PMLiVE

CHAPLE disease

- 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

- 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

FDA authorises first at-home COVID-19 and flu test for emergency use

The single-use test uses nasal swab samples and provides results in about 30 minutes

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

Pfizer’s maternal RSV vaccine candidate accepted for FDA priority review

Globally, RSV infections cause over 100,000 child deaths annually

- PMLiVE

FDA external report recommends actions to address US opioid crisis

Over 100,000 Americans died from a drug overdose in 2021, with 70,000 of those due to opioids

- PMLiVE

FDA panel backs over-the-counter use of Emergent BioSolutions’ Narcan

The nasal spray works by blocking the effects of an opioid overdose if administered in time

Biogen Idec building

Safety-related change to label for Biogen’s Alzheimer’s drug Aduhelm approved by FDA

Larger areas of bleeding on the brain have been reported in some patients taking the drug

- 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

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