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'This means the world to us'... parents hail offer of new drug to help ill children

By Eimear Rabbitte

The parents of three young children suffering from a rare genetic condition have spoken of their relief after they were finally granted access to a potentially lifesaving drug.

Mia Warren (6), Caoilte Fitzsimons (3) and Noah Collins (3) are three of only four children in Northern Ireland who suffer from Type 1 spinal muscular atrophy (SMA). SMA is a genetic disease that affects motor neurons, causing progressive muscle weakness and loss of movement.

All three children have very limited movement, while Caoilte requires round-the-clock ventilation due to the condition, which can also cause severe respiratory difficulties.

The life expectancy of babies with SMA Type 1 is typically only eight months, with most not living past the age of two. However, Mia, Caoilte and Noah have all managed to defy the odds.

This week their mothers spoke of their frustration that their children had yet to be allowed access to a drug called Nusinersen, which saw positive results during clinical trials in the US.

It was approved by the FDA in the States on December 23 and while it has yet to be authorised by the European Medicines Agency (EMA), the drug has been made available free of charge via an Extended Access Programme at the Royal Victoria Hospital, Belfast. However, only one other child with the condition in Northern Ireland had started treatment at the hospital.

The families said they had been told resource and space difficulties were preventing them from accessing the programme. After speaking to the families this week, the Belfast Telegraph contacted the Belfast Trust and Department of Health for comment and later received a statement from minister Michelle O’Neill confirming that the children had now been allowed on to the programme.

Mia’s mother Lisa Warren said they were “over the moon” after being contacted personally by the Health Minister.

“Shocked isn’t the word, it all came completely out of the blue. I nearly had a heart attack.

“It was such a turnaround... it just means the absolute world to us,” she said.

“It comes from all three families really pulling together to get our children this treatment because it is a lifesaving drug.”

It was an extra special moment for Lisa and her partner Stephen from Holywood, as the parents of little Caoilte — Rob and Fiona Fitzsimons — happened to be in the house with them at the time of the call. The drug is likely to see massive differences in the quality of life for Mia, Caoilte and Noah.

North Belfast youngster Caoilte, who turns four this June, requires breathing aid from a tube into her trachea at all times.

“But her mind is very active,” Fiona said.

Noah from Lisburn turned three this month and is a “very happy little boy”.

Currently, he has very little head control and only slight movement in his legs. His father Jonny gave up work to become Noah’s full-time carer.

“When he is well he just melts your heart,” mum Rachael said.

All three families attended a meeting with the Belfast Trust yesterday. The trust is currently working on securing theatre space and paperwork from drug company Biogen before confirming the start date for treatment

Mrs O’Neill said she was “absolutely delighted” with the outcome.

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