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Wakix

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BeiGene’s Brukinsa receives CHMP recommendation for chronic lymphocytic leukaemia

CLL accounts for around one-quarter of new leukaemia cases

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

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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

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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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Pfizer/BioNTech BA.4/BA.5 COVID-19 booster receives CHMP recommendation

If an authorisation is granted, doses of the bivalent booster will be available to all 27 EU member states

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Bristol Myers Squibb’s Opdualag approved by CHMP for melanoma

If approved by the EC, Opdualag would be the first LAG-3 blocking antibody combination available in Europe

- PMLiVE

AstraZeneca’s Tezspire recommended for approval in the EU by CHMP

The recommendation is for the treatment of severe asthma as an add-on therapy for patients 12 years and above

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Gilead’s COVID-19 drug Veklury recommends for approval by CHMP

If approved by the EC, Veklury will become the only direct-acting antiviral with full marketing authorisation in the EU

- 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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