Research News

Pharmaceutical company PepGen has published preliminary results from the first phase of its FREEDOM2-DM1 clinical trial, which is investigating a potential new therapy for people living with myotonic dystrophy type 1 (DM1).

FARA Ireland's April 2026 Advocacy Bulletin details the latest steps being taken to secure reimbursement of SKYCLARYS for people living with Friedreich's Ataxia in Ireland. Download the full bulletin for an update on where things stand.

Muscular Dystrophy Ireland warmly welcomes the introduction of testing for spinal muscular atrophy (SMA), commencing today as part of the expansion of the National Newborn Bloodspot Screening Programme

In November, MDI, in collaboration with Duchenne families, launched a survey to capture the lived experience of families living with Duchenne muscular dystrophy to support the patient submission for the reimbursement of givinostat. We would like to say a heartfelt thank you to everyone who took the time to participate.

This project forms part of a wider research study examining the social and economic impacts of living with a rare disease on families of children with rare diseases across Ireland and Northern Ireland. The research is being conducted as part of a PhD project by researchers at University College Dublin.

A review is under way to decide if Givinostat for Duchenne muscular dystrophy will be reimbursed in Ireland. Your experience of living with Duchenne can help inform this decision - share your story in the survey link.

Have your say on what matters most when accessing and using community-based healthcare services. A PhD Researcher in Health Economics at University College Cork, is exploring how community-based healthcare services can better support people living with neuro-physical disabilities. The results will give a clearer picture of how to design community-based care that truly meets the needs of people with neuromuscular and other neuro-physical conditions. This evidence will help guide public policy, reduce inequalities, and improve social wellbeing and sustainability.

As part of IPPOSI’s Patient Capacity Building Programme, IPPOSI is delighted to host an online information webinar in collaboration with ClinicalTrials.eu – a new European Union platform designed to make clinical trial information more accessible to the public.

Please join us in asking your local TDs and senators to keep this matter on the political agenda by attending a briefing on the campaign at the Audio-Visual Room, Leinster House at 2.30pm on Wednesday 11 June. and leveraging your role as you elected representative to highlight the importance of timely and planned action. Time is muscle and delays in reimbursement risk excluding eligible children simply because the disease progresses while they wait.

Duvyzat™ receives positive recommendation from the EMA’s CHMP for conditional approval in DMD.
Click to read what this could mean for families and what happens next.