HSE Patient Feedback For National Genetics And Genomics Office
National Genetics & Genomics Office are aiming to better understand what people who are referred to genetic and genomic services, and their families, need to know to help them better navigate services. The knowledge gained will be used to work towards an improved patient experience of using genetic and genomic services. More information the advert attached. Expressions of interest close on 23rd February 2024.
EMA confirms recommendation not to renew marketing authorisation for Translarna
The CHMP agreed to re-examine its decision on request of the manufacturer PTC. This took place in January and despite efforts from the manufacturer and patient community the EMA announced on Friday, 26 January 2024 recommendation for non-renewal of authorisation of Translarna. The process found that while there was evidence of benefit from the treatment, it’s not statistically significant. Importantly there was no concerns about the safety of the treatment.
European Reference Network – Patient reported outcome measure.
The Patient Advisory Board of the European Reference Network on Neuromuscular Diseases (ERN EURO-NMD) have prepared the following survey for adults (aged 18 and over) living with a neuromuscular condition. The purpose of this survey is to identify the types and severity of symptoms individuals experience
State of Caring 2024 – Family Carers Ireland
On behalf of Family Carers Ireland, you are invited to participate in their State of Caring 2024 survey. This biennial survey is a vital initiative aimed at comprehensively understanding the lives and experiences of family carers across Ireland who are currently providing care. Previous reports have played a crucial role in shedding light on the impact of caregiving.
Expression of Interest - MDI Research Committee
Two positions have become available on the Muscular Dystrophy Ireland (MDI) Voluntary Research Committee. We are seeking expressions of interest for a person with lived experience of neuromuscular conditions. This includes people living with a neuromuscular condition, or a family member of a person living with a neuromuscular condition. We also welcome expressions of interest from health professionals, researchers and others who are working in or have an interest in contributing and partnering in the development of MDIs research strategy.
Round Up On Research Developments By Condition
Neuromuscular conditions can be difficult to diagnose and manage, so research is vital for families and for quality of life. In this issue, we include a round-up of some research developments that have taken place in recent months
Transitioning To Adulthood
Are you over 18 years of age, living with a neuromuscular condition and interested in sharing your experiences and challenges of transitioning to adulthood such as leaving secondary school, getting a job, going to post-secondary education, meeting young people socially etc?
Building Connections Programme
The Building Connections programme was a research initiative facilitated by PPI (public and patient involvement) Ignite Network in collaboration with MDI. This programme ran over three weeks from 31 October to 14 November
Call For Participants: Take part in Duchenne Muscular Dystrophy (DMD) Study
Calling adults with Duchenne Muscular Dystrophy to share their experiences of moving into adulthood.