Recently, families have been given hope of accessing a life-changing treatment on the NHS, which can help slow down the progress of a rare muscle-wasting condition.
Across the UK, 1,300 people are affected by a rare disorder called spinal muscular atrophy (SMA), symptoms of this condition mean it can cause irreversible loss of children’s ability to walk, crawl, swallow and breathe.
According to charities Muscular Dystrophy UK and Spinal Muscular Atrophy UK, Spinraza is one of the first drugs to hopefully be approved and is expected to be routinely available for people with SMA Types 1, 2, and 3 from April 2019.
Eilidh, 10, has SMA type 2, her mother, Dr Sheonad MacFarlane said: “Having access to Spinraza means so much to us and to other families.
“We can now look to a future where those living with SMA can have a better quality of life.”
Amy Cameron’s three-year-old son, Zac, has SMA Type 1 and also has access to Spinraza on the NHS.
Mrs Cameron said: “Our whole life was thrown into a whirlwind the day we heard those three words – spinal muscular atrophy.
“But Spinraza has bought us valuable time with our little boy and given us hope for the future. Zac has achieved things we never thought possible and continues to amaze us every day.”
Muscular Dystrophy UK has called for wider access to the treatment to be introduced across the UK.