The parents of a child with a rare muscular disease are urging that the State does "not delay any longer" in making decisions on access to a drug that has been approved for use in Europe but has not been approved for reimbursement in Ireland.

Patients with rare diseases and their families travelled to Leinster House to call for action on access to new medicines.The group is calling on the Government to introduce a dedicated new line item in the Budget for new drugs, a review of the reimbursement scheme and an expansion of the Early Access Programme.

Rare Diseases Ireland is testing a new questionnaire designed specifically to capture the healthcare experiences of people living with rare diseases and their caregivers. The questionnaire was built specifically for rare diseases - covering the things standard questionnaires always miss, like how long it took to get a diagnosis, or what it's like coordinating care across multiple specialists.