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- PMLiVE

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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

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

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

Ipsen to acquire rare disease specialist Albireo in deal worth over $950m

The deal will strengthen the biopharma’s liver disease pipeline with the addition of Bylvay

- PMLiVE

Amgen announces plans to acquire Horizon for $27.8bn in biggest pharma deal for 2022

The acquisition will see Amgen pay $116.50 for each Horizon share in cash

- PMLiVE

Alexion’s Ultomiris shows promise in neuromyelitis optica spectrum disorder phase 3 trial

Ultomiris-treated patients showed zero relapses with a median treatment duration of 73 weeks

- PMLiVE

FDA awards over $38m for advancement of rare disease treatments

Grants will support clinical trials, natural history studies and regulatory science tools

Patient Centric Approaches for Rare and Ultra-Rare Diseases

Lawrence Bressler, Head of Value & Market Access, Global Rare Diseases at Chiesi, shares how Chiesi is revolutionizing the lives of people living with rare diseases on a global scale....

Impetus Digital

- PMLiVE

Reframing rare to increase earlier diagnosis of Gaucher Disease

Sanofi Genzyme wanted to reach haematologists; the specialist physicians most likely to encounter patients with Gaucher Disease prior to diagnosis. The problem? Gaucher Disease is considered very rare and its...

Create Health

- PMLiVE

Rare diseases: making a difference through education

Medscape Education team up with The Guardian to launch a Rare Disease campaign in the UK.

Medscape Education Global

- PMLiVE

BeiGene’s Brukinsa gets green light from NICE as Waldenström’s macroglobulinemia treatment

The condition occurs in less than 2% of patients with non-Hodgkin lymphomas

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