Lewis Harte Walsh with his parents, Senator Rose Conway-Walsh and a group of supporters outside Leinster House.

Families in Dublin to urge u-turn on life-changing drug

YESTERDAY, families met with Minister for Disabilities Finian McGrath and Deputy Mary Lou McDonald in Leinster House to discuss access to the life-changing drug Translarna, a treatment for boys with a specific type of Duchenne Muscular Dystrophy (DMD).

Mayo boy Lewis Harte Walsh is one of five boys in the Republic of Ireland fighting for access to the drug that could help them keep walking.

Muscular Dystrophy Ireland (MDI) and the families have called on the HSE to reconsider its decision announced last month to not fund Translarna.

This latest lobbying meeting was facilitated by Senator Rose Conway-Walsh.

Anne Marie Harte, mother of five-year-old Lewis, from Castlebar, said he son is eligible for the treatment, adding: “It is so unfair knowing that there is a treatment out there that can help our son but it is being denied to him. This is a race against time that we don’t have. it’s simply agonising.”

Translarna slows down the progress of DMD, that sees young boys losing the power of their muscles and being consigned to a wheelchair in mid childhood.

Dr. Declan O’Rourke, consultant paediatric neurologist, Temple Street Hospital, confirmed: “Treatment with Translarna may change the course of the disease and help delay key milestones, potentialyl improving an overall life expectancy of patients. Considering the high unmet need, the burden of Duchenne Muscular Dystrophy on society and the clinically meaningful trial use of Translarna and its well-tolerated safety profile it was very disappointing to hear of the HSE Drugs Group decision not to reimburse this treatment.”

He added: “l hope that the meeting yesterday will help provide a pathway for a successful outcome for the boys and families at the heart of the issue.”

Translarna has been reimbursed in the UK for over a year now.

The Irish families have called on the Minister for Disabilities to intervene and ensure their sons receive treatment without further delay and are given the same opportunities as other children across Europe – the treatment is available in over 20 countries in Europe.

Not only will this drug enable the boys to participate for significantly longer as an active member of society, it also delays the requirement for other medical interventions and extremely expensive transport and social care supports.

DMD is the most common form of muscular dystrophy and it’s estimated there are around 100 boys and men living with it in Ireland.