Colchester mum's battle to get son dose of £1.7m drug

Megan Willis’ eight-month-old son Edward is on the waiting list for a special medicine called Zolgensma.

The drug, which costs roughly £1.7 million, has just been approved for use in the UK following more than three months of trials.

It is used to target the root cause of, and slow down the impact of, a rare condition called Type 1 Spinal Muscular Atrophy, which Edward is battling.

About 40 children are born with the most severe form of the disease every year and they rarely live beyond the age of two if they do not receive the correct treatment.

Previously the drug was unavailable on the NHS and could only be administered privately at an eye-watering cost.

This week, however, a five-month-old boy called Arthur, from London, became one of the first to receive a dose.

The NHS roll-out has provided Edward and his parents with a degree of hope, but because of his age he could now be waiting months before he gets the drug.

“It is really frustrating because they have started giving the treatment to children under six months, but Edward is now eight-months-old,” added Megan.

“We are told he will get it but it has been a nightmare and we are still trying to fight it because he could be waiting months.

“That could be detrimental to Edward’s development because time is of the essence, yet we have not been told anything.”

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Edward was born last September at Colchester Hospital but had to go to Addenbrooke’s Hospital in Cambridgeshire.

He was diagnosed with Type 1 Spinal Muscular Atrophy seven weeks later.

The genetic neuromuscular condition causes progressive muscle wasting and weakness which ultimately leads to a the loss of movement.

Edward’s ability to crawl and walk, and move his arms, hands, head, and neck will gradually deteriorate and his breathing and swallowing will become difficult.

Since his diagnosis, Megan, 30, of Colchester, and partner John Hall, 37, have been trying to raise £1.2 million to purchase Zolgensmas privately.

Megan has now questioned why it has taken so long for the drug to be made available.

She added: “The NHS is an amazing institution, but children with SMA do not have time and I feel like they have been let down.

“Edward is responding really well to the treatment he is on, but it is not as good as what we could be getting.

“The younger they are when they get the drug, the better chances they have.

“Until Edward gets the drug we will be anxious, because I worry, is he getting pushed down the line every time a new baby is born?”