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- PMLiVE

genetic diseases

- PMLiVE

Medscape Education and Medics4RareDisease Uncover the Education Gaps in Their Compelling Rare Disease Day Campaign

Medscape Education and Medics4RareDisease are partnering on a Rare Disease Day awareness campaign. The campaign will form part of a series of short programs produced by ITN in collaboration with Genetic Alliance...

Medscape Education Global

- 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

- PMLiVE

EFPIA-EURORDIS joint statement published on patient access to medicines for rare diseases

The statement contains a series of proposals to improve the lives of people living with rare diseases by addressing access barriers to transformative medicines

- PMLiVE

Novo Nordisk and Flagship Pioneering to collaborate on new research programmes

The companies will focus on rare and cardio-metabolic disease areas

- PMLiVE

Nucleus X Consulting – where patient engagement meets strategic consultancy

Nucleus X Consulting consists of an agile team of specialists that combine expertise in all aspects of patient engagement along with strategic consultancy in rare disease and early-phase medical commercialisation.We...

Nucleus Global

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