Help Toby on his battle with Propionic Acidemia

Hi , As most of you know my daughter Lucy and her husband had their baby boy Toby 7 weeks ago who was diagnosed with Propionic Acidaemia. (PA) is a rare inherited metabolic disorder, which affects the protein pathway (can affect heart, brain, bone marrow etc.). It is a lifelong, life-threatening condition. The main complications associated with propionic acidaemia include recurrent episodes of metabolic decompensation with illness or fasting, cardiomyopathy, movement disorders, bone marrow suppression and recurrent pancreatitis

Currently they are battling with the hse to supply Toby with a life saving drug called Carglumic acid.

This drug if approved will hopefully keep toby stable at home while they are working on long term treatment options .

The process has been started for liver transplantation which unfortunately is not carried out on the Island of Ireland , this will mean frequent travel and a long stay in London .

The other option currently being explored is clinical trial MRNA therapy based in Manchester.

Both of these treatment options will be time consuming but also extremely costly

If anyone had the ability to financially assist Toby on his battle we as a family would be extremely greatful

Thank you ❤️