Save baby Mariam’s life!

Baby Mariam is 3 years old and was born on 7th June 2020 in Babylon, Iraq. Mariam is the youngest of the family and it took her parents 7 years to conceive her. Unfortunately, Mariam was born with a rare genetic condition called spinal muscular atrophy (SMA), which causes muscle wastage and weakness. At 4 months old, doctors in Iraq suspected Mariam had SMA Type I; the most severe type that usually does not allow children to live beyond the age of two (as their muscle weakness eventually affects the important muscles needed for breathing). 

Mariam is still alive because of the medication she is receiving; however, it is an expensive treatment which must be taken for life. 

Thankfully, Mariam’s parents took her to the UAE to undergo genetic tests & was seen by specialists who confirmed her diagnosis and that she is fit to receive the life-saving injection; Zolgensma.

Prior to having the injection, children need to have an antibody test to check their suitability – Baby Mariam has had this done already in the UAE and is suitable for the treatment. 

The sooner the treatment the better the outcome.

 Treatment Options 

There are other treatments available for those diagnosed with SMA I and they can be seen in the table below. 

Whilst Spinraza is the oldest treatment within the medical field, it is a life-long form of treatment which would leave Baby Mariam dependent on the injections for the rest of her life. 

Risdiplam has similar issues, with it also being a life-long treatment and in the long run would be much more expensive for Mariam’s family at a staggering $8000 a month. 

Zolgensma, whilst a high initial cost in the medium to long term, it is the most cost-effective treatment option, ensuring a one-off treatment unlike the others, preventing the disease by replacing the gene that does not work/is missing. 

About Zolgensma 

Zolegensma is infamously known as ‘the worlds most expensive drug’ was first trialled in 2017 and since then has had 3 more trials conducted, all with good results deemed “effective at saving the lives of children”. 

The injection has been approved by the Food and Drug Administration (FDA) and the National Institute for Health and Care Excellence (NICE) and has been used by the NHS for children under 12 months since March 2021. 

Ideally, the injection treatment should be accompanied by physiotherapy and blood tests to monitor the liver on a weekly basis over three months. 

Prior to having the injection, children need to have an antibody test to check their suitability – Baby Mariam has had this done already in the UAE and is suitable for the treatment.