US drug maker sues to force Ireland to offer expensive Duchenne’s treatmentBMJ 2017; 358 doi: https://doi.org/10.1136/bmj.j4302 (Published 15 September 2017) Cite this as: BMJ 2017;358:j4302
- Owen Dyer
The American pharmaceutical firm PTC Therapeutics is suing Ireland’s Health Service Executive (HSE) over its decision not to fund drugs for two boys with Duchenne muscular dystrophy (DMD).
PTC’s drug, ataluren (Translarna), has been approved by the European Medicines Agency (EMA) to treat the nonsense mutation form of DMD in children aged over 5. This condition affects two boys over 5 in the Republic of Ireland and one in Northern Ireland. The boy in Northern Ireland is receiving the drug and has benefited, according to his parents. The two boys …