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Compassionate use programme

- PMLiVE

UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

- PMLiVE

Turn payer insights into impact with omnichannel engagement in market access

By Betty Charleshouse

Amiculum

- PMLiVE

Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

Photo of Tim Wright plus title of talk

MFN Pricing Is Moving Fast – Here’s What Matters

With GENEROUS, GUARD, and GLOBE all released since December, 185 pages of policy will materially impact U.S. drug pricing — and yes, it's a lot to digest. In a new...

Genesis Research Group

- PMLiVE

ISPOR’s 2026–2027 HEOR Trends report: The Era of Evidence Judgment

In response to ISPOR’s recently published 2026-2027 HEOR Trends, David Miller, CEO, Genesis Research Group, suggests that we’re no longer being evaluated on our ability to produce data, but on...

Genesis Research Group

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

Headshot of Sarah Theobald with text reading 'Healthcare communications trends in 2026'

What will healthcare communications look like in 2026? Amiculum’s prediction of upcoming trends

By Sarah Theobald

Amiculum

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

Symptoms of the rare disease include bleeding, eczema and frequent infections

- PMLiVE

Does real-world use of AI in systematic literature reviews result in time and cost savings?

By Manca Povsic and Emma Griffiths

Amiculum

- PMLiVE

AstraZeneca announces $2bn investment in US manufacturing

The new and expanded facilities will support production of rare disease drug treatments

Quote from the article: Without adapting JCA to include assessment of real-world data and broader benefits, vaccines may be undervalued

Is Joint Clinical Assessment (JCA) appropriate for assessing the broader value of vaccines?

By Emily Morton-West

Amiculum

- PMLiVE

AstraZeneca reports positive results from rare disease trial

HES is a group of rare disorders characterised by persistently elevated levels of white blood cells

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