Wonder drug gives hope to families of sick children

Steven Boobyer and Lisa Warren with their daughter Mia

Steven Boobyer and Lisa Warren with their daughter Mia

A Belfast mother is hoping that a new wonder drug being trialled in Northern Ireland can help her sick daughter.

Mia Warren was diagnosed with Spinal Muscular Atrophy (SMA) type one at the age of seven months. Until the end of last year, her mum Lisa said the condition was untreatable, however a scientific breakthrough has offered hope for her little girl, now six.

Nusinersen, hailed as a wonder drug in America, is being trialled at Royal Victoria Hospital and Mia is one of three Northern Ireland children with SMA whose families are calling on the Belfast Health Trust to explain why they are being denied access to the drug.

A fourth child has been receiving the unlicensed drug, the health minister confirmed.

Mia’s mother Lisa said: “It’s so frustrating to have this drug in our grasp, something that could help Mia, but we can’t access it.”

SMA affects Mia’s crawling, walking, arm, hand, neck and head movement, breathing and swallowing.

Lisa said: “Our daughter is the absolute light of our lives. She is bubbly, bright and full of mischief despite her condition. However, every winter brings extra worry in case Mia catches a cold. Only recently we were with her in hospital watching helplessly while she fought through pneumonia.”

A trust spokesperson said: “There are strict criteria applied to unlicensed drugs and they follow a strict and complex approval, based on clinical need.”

Health Minister Michelle O’Neill said last week there had been a “breakdown in communication” and all families would be offered a face-to-face meeting with clinicians who can discuss their child’s suitability for trial.