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The EMA launches a public consultation on registry-based studies
A few weeks ago we published a blog explaining what a patient registry was. These databases enclose quantitative and qualitative data about patients who affected by a particular condition, like for -
Lessons learned: Share4Rare’s new Digital Informed Consent
All around the globe, 2020 has brought with it a lot of downs. However, it has also brought some very valuable ups, mainly in the form of lessons learnt and powerful insights into what we are capable -
The case of undiagnosed profiles in Share4Rare
Share4Rare has very nice support for patient profiles with a diagnosis. These users will find that Share4Rare is a system in which they can interact, communicate, and share knowledge with other users -
Gathering people’s knowledge on cell and gene therapy to create tailored educational materials for patients and citizens
Being educated on health sciences can bring you numerous benefits as a regular citizen, especially if your are a patient or a caregiver: this way you will be more empowered on and will be able to -
The MRC Centre for Rare and Neuromuscular Diseases Biobank
The principal objective of the MRC Biobank is to collect biomaterial from patients affected by neuromuscular disorders with the aim of supporting translational research. The availability of high -
World Duchenne Awareness Day: "Together, we are stronger"
This year's awareness campaign ‘Together, we are stronger’ was translated in 15 languages and collected over 1,000,000 views globally. This impressive number was reached due to an amazing -
Data for a difference — The Share4Rare research feature for patient organisations
Violeta Astratinei is a Ph.D. biologist with a background in environmental research. After she lost her sister to melanoma in 2014, she became a core member of Melanoma Patient Network Europe, MPNE -
Gemma Marfany: "Researchers, patients and families must come together to fight rare diseases"
Gemma Marfany is a professor in the Department of Genetics at the University of Barcelona (UB) and head of Unit U718 at CIBERER, the Spanish biomedical research centre for rare diseases. She is also a -
EURO-NMD – An overview of European Reference Network for neuromuscular diseases
A staggering 7000-8000 rare diseases affect the daily lives of around 30 million people across the European Union. Even though EU citizens benefit from improved access to healthcare throughout the -
New Share4Rare webinar: “International patient registry for rare diseases and COVID-19”
On September 18th Share4Rare organizes a webinar along with the Garrahan Hospital from Buenos Aires (Argentina) to present the new international patient registry that will be created in the platform -
World Duchenne Awareness Day 2020: Duchenne and the brain
Learning and behavioural challenges in DMD and BMD Both conditions are characterised by progressive muscle breakdown. A certain part of the DNA is missing, duplicated, or changed so the code cannot be -
Between hype and hope: the truth about gene therapy and genome editing in Duchenne muscular dystrophy
About Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy affecting around 1 in 5,000 newborn boys worldwide. The main characteristic is a