On Sunday, September 15th, Smith & Brock will be taking part in the London to Brighton bike ride to raise funds for our very own Steve and his twin boys, who suffer from Spinal Muscular Atrophy type 1 (SMA 1) disease.

We would really appreciate it if you could help support Steve and his family. Even the smallest amount will help contribute towards their specialist equipment and therapy needs to give Raffy and Siddy the best quality of life.

As a registered charity, Tree of Hope are able to help them raise more money for Raffy and Siddy as Gift Aid can be claimed on each donation, maximising the fundraising total.

Tree of Hope will manage Raffy and Siddy's fund and pay invoices directly, and therefore donors can be rest assured that all money raised is being spent directly on the needs.

Please remember to tick the Gift Aid box when donating, thank you.

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Meet Raffy and Siddy

Steve & Jenna's identical twin boys...

One day after their 1st birthday, they were diagnosed with Spinal Muscular Atrophy type 1 (SMA 1), a rare progressive neuromuscular genetic disease that, if left untreated, is fatal before 2 years old. Steve and Jenna noticed that the boys were not meeting typical milestones like holding their heads, rolling, and sitting unaided. They also had a constant rattly chest that couldn't clear.

Their Diagnosis

Jenna took them to the doctors, hospital and contacted the health visiting team, but each visit ended with the same outcome: being told that they would catch up and that they were lazy because they are twins, and Jenna even got told that she was just being neurotic.

Raffy and Siddy were given lots of antibiotic courses to clear their chests, but none of them worked as their chesty rattle was due to respiratory weakness and their lungs were deteriorating, a common symptom of SMA 1. After being knocked back by medical professionals for 10 months, they finally got a GP to take them seriously, referring them to the paediatric consultant at the local hospital, only to be told there was a 6-month waiting list. Luckily, Jenna fought hard for an earlier appointment; otherwise, they may not have been here to tell their story given that SMA 1 is fatal before 2 years old. At that appointment, they had genetic testing and were informed of the devastating results a few weeks later.

Lifesaving Treatment

The twins have been extremely fortunate that the NHS has provided them with Gene Therapy using a (fairly) new drug called Zolgensma. Zolgensma targets the genetic root cause of SMA by replacing the function of the missing SMN1 gene with a new, working copy.

Any cells that have already died can't be replaced, but going forward, any remaining cells should survive, function, and be maintained. As it is a new drug and they are the first generation of children to receive this treatment, there is no data past 6 years of age, but those that have received this drug are alive and doing well. Mobility issues are still present, and it is likely they will be in wheelchairs for life. But there have been many gains, and some children have even hit new milestones. Historically, this is something that never happens with SMA 1, as it is a progressive disease.

Steve and Jenna are beyond grateful to the multidisciplinary team panel at the NHS, who decided they were worth saving. Zolgensma has given them a second chance. However, Zolgensma without physiotherapy was described as a 'gun without bullets'. They will require a lifetime of extensive physiotherapy, hydrotherapy, specialist equipment, and home adaptations to help them become independent and lead the best quality of life, which the NHS just doesn't have the funding for.

Please support Steve and his family to have the best chance at meeting new milestones. Zolgensma has given the twin boys a second chance and they need to take every opportunity that they can to excel with this new drug.