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rare diseases

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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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

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

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

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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

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Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

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FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

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AstraZeneca announces $2bn investment in US manufacturing

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

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AstraZeneca reports positive results from rare disease trial

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

Genesis giveaway of a minifigure

Genesis Research Group contributes strong scientific program to ISPOR Europe and warmly invites visitors to booth 212

Genesis Research Group is proud to be attending ISPOR Europe 2025 in Glasgow from 10-12th November with a strong scientific program that includes an HEOR Theater session, contributions to 10...

Genesis Research Group

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Insights From ISPOR 2025: Breaking the Data Barrier in Rare Diseases – Can AI Bridge the Gap?

Last month, the ISPOR annual meeting in Montreal was brimming with opportunities to explore the latest developments and insights in health economics and outcomes research (HEOR) and market access. In...

Petauri Evidence

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Insights From ISPOR 2025: R In Health Technology Assessment – Is It The Future?

ISPOR’s annual meeting in Montreal this May was packed with opportunities to explore the latest trends, insights, and research in HEOR and market access. In this series, our experts share...

Petauri Evidence

ISPOR partner seal

Genesis Research Group announces inaugural membership of ISPOR Corporate Partnership Program

Genesis Research Group, a pioneer of tech-enabled real-world evidence (RWE), health economics and outcomes research (HEOR), and market access services, today announces it has joined selected organizations as an inaugural...

Genesis Research Group

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