Hannah is a very special little girl who at 15 weeks old was diagnosed with a rare muscle disorder called Spinal Muscular Atrophy (SMA). Hannah has type 1 of the disease which is the most severe.
SMA is a genetically inherited condition that is caused by a faulty gene, and affects the muscles in the body. Approximately 1 in 10’000 babies per year in the UK are born with SMA.

SMA is a degenerative, life limiting condition. Without treatment babies with type 1 rarely see their 2nd birthday. Until recently there was no treatment for SMA, however in the past few years, there have been advances in medical technologies and research, and thankfully there are now treatments available for this condition.

In February 2021, thanks to the hard work of doctors at Ninewells Hospital Dundee and the Royal Children's Hospital Glasgow, Hannah became the 1st child in Scotland to be given a drug called Zolgensma, on an unlicensed basis.

This treatment is a brand new Gene Therapy, which gives the patient an active copy of the faulty gene which causes SMA. This is embedded in a Virus and infused intravenously over 1 hour to ensure it reaches all parts of the body.

Hannah is now 1 year old and 9 months post infusion and although she is making progress, she still faces many challenges. The areas of improvement that have been noticed are increased head control, improved upper body strength, where Hannah is now able to grasp and play with small, light objects and toys. Hannah continues to be an oral feeder, which can be challenging for children with SMA Type 1.

Although these are significant achievements for Hannah her journey so far has had many challenges. This has meant several stays in Ward 29, Ninewells hospital to fight off chest infections, particularly as the seasons have changed and winter bugs have been on the increase. Hannah has recently visited the Royal Childrens Hospital, Glasgow, to establish her on a non-invasive ventilator to assist with her breathing at night, and to try and ward off future chest issues. She also requires 24 hour postural support to prevent scoliosis which is a condition common with SMA type 1 children. Postural support also helps Hannah to engage with toys, food and the world around her.

Hannahs needs are continually changing, especially as she grows and develops. We have already made several changes to support Hannah with everyday life, for example making the front of our home wheelchair accessible, and purchasing some supportive seating to allow Hannah to attend Nursery and engage with other children her age. Any charitable donations made for Hannah will be put to good use to provide her with a good quality of life, and the same oppurtunities as any other child her age.

Hannah has had some major challenges in her life so far, and will have more in the future, however she is full of giggles and smiles and loves to play and interact with everyone she meets.