This page has been set-up to raise funds for our son Remme who was born on 1st March 2021.

On December 23rd 2021 our world was turned upside down. Our beautiful 9 month old baby boy was diagnosed with Spinal Muscular Atrophy Type 1 (SMA) a rare genetic condition that progressively destroys motor neurons causing muscle weakness and atrophy (when muscles get smaller).

 

Having never heard of this before, with no known family history we began our research, discovering that this DNA related disease robs many children of their ability to reach developmental movement milestones; sitting up, crawling, walking and eventually failing their capabilities to eat and breath, taking away their chance of life before they reach the age of 2 years old.

 

Thankfully, only last year, a lifesaving treatment has become available for these incredibly vulnerable young children and our Remme is eligible to receive this treatment; a gene therapy treatment that is reportedly “The most expensive drug in the world” costing £1.8 Million. However, it breaks our hearts to say, that this is no cure.

 

Zolgensma is the first gene replacement therapy treatment that delivers functional copies of the essential gene SMN 1 to help build muscle movement.

Zolgensma without physiotherapy is like a "war without guns". Remme will need extensive physiotherapy/ hydrotherapy 3+ times a week and an array of specialised equipment to get the very best results from this treatment. This level of therapy could be the difference between Remme crawling, and not crawling, walking and not walking, fighting and being defeated.

 

We are reaching out to our family, friends and anyone with kindness in their hearts to help us raise funds to give our son the best chance of challenging this disease following his life saving treatment, giving him the freedom in life that he deserves.

 

We have a long road ahead of us with a lot of uncertainty, with many ups and downs, struggles and success. But Remme is brave, strong, determined and blessed.

 

Remme will be receiving his treatment on Tuesday 11th January 2022, an incredible 18 days from being diagnosed.

We cannot be thankful enough to have been given the opportunity to receive this gene therapy treatment, to all those who helped produce it, to all those brave children and their parents who enrolled in the trials to build the success in this drug, to the incredible NHS team who recognised this devastating disease in our boy and accelerated through the necessary steps to start him on the road to recovery.

 

We live in a cruel world sometimes, but a world full of many incredibly intelligent and selfless people who dedicate their lives to help others.

 

 

Please share and join us in Remme’s fight to recovery and to help him live his life like any other ‘Cheeky Chappy’ his age.

 

 

 

 

 

Thank you for your support

Scott Leah Harley & Remme