A three-year-old girl born with a severely life-limiting condition has for the first time moved independently, just two weeks after she started taking a miracle drug. Caoilte Fitzsimmons, who has Type 1 Spinal Muscular Atrophy (SMA), was previously unable to take Nusinersen as it was unavailable in Northern Ireland. An extended access program was launched allowing one lucky child in Belfast to undergo the treatment and this was later extended to help two other children, including Caoilte.
SMA restricts muscle movement and sufferers need constant care full-time. But just a fortnight in to the treatment, Caoilte is now able to hold her head up independently and move her feet, toes, arms, and legs and before she couldn’t. Her dad, Rab, admitted that the significant changes in such a short period of time have made him feel emotional. She had her second treatment on Tuesday and the progress so far has been wonderful so they are hopeful that things keep will improve
Mia Warren, six, and Noah Collins, two, are also receiving the miracle treatment, which was previously only available in the United States. Mia was already able to move before starting the treatment but her mum, Lisa, has noticed that she is getting stronger. She wears a body brace, and she was never able to sit up without it, but a couple of days ago she sat for a minute without being held. She was also able to turn her head more as well.