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patient–HCP communication

- PMLiVE

LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

- PMLiVE

Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

- PMLiVE

PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

Involvement of children and young people in research and publication development

From ‘why’ to ‘how’: patient partnership across age groups in research and publications

By Sarah Mohamad

Amiculum

phthalmologist, optometrist checking child, little girl vision looking for problems and caring for eye

Why patient engagement must come first in rare disease trials

Explore why patient engagement is the cornerstone of successful rare disease trials, emphasising the importance of designing studies around the realities of patients' lives to build trust and improve outcomes.

Cuttsy + Cuttsy

Fan image of Site Report

New Cuttsy+Cuttsy research shows why site support matters more than ever

Cuttsy+Cuttsy's upcoming report, 'The site factor: Where protocol meets practice in clinical trials', delves into the critical role of site staff in clinical research, offering insights into their daily challenges,...

Cuttsy + Cuttsy

A quote that reads 'early-stage planning is not just helpful - it's a survival tool

Winning in Pharma: the power of early strategic planning with patients

By Zoë van Helmond

Amiculum

- 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

Sanofi announces positive results for venglustat in type 3 Gaucher disease

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

Amiculum meets Trishna Bharadia, patient engagement and health advocate

How can pharma build genuine partnerships with patient communities?

Patient engagement is not optional in pharma – it’s integral. Meaningful partnerships with patients, caregivers and advocates must shape every stage of healthcare and medicines development, driving better outcomes, equity...

Amiculum

- PMLiVE

Havas Life London unveils AI awareness campaign

The campaign highlights the dangers of AI misinformation in rare disease

- PMLiVE

FDA approves first gene therapy for Wiskott-Aldrich syndrome

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

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