This page has been set-up to raise funds for our son Edward who
was born on 7th September 2020 and diagnosed with Spinal Muscular
Atrophy (SMA) type 1, at 7 weeks old on 29th October.
Our world has been turned upside down, in the most extreme way.
Edward has brought purpose to our life and makes each day shine in
the brightest way possible. He is the most beautiful, happy baby
and it breaks our heart that he has this condition.
Spinal Muscular Atrophy (SMA) is a rare, genetic neuromuscular
condition causing progressive muscle wasting (atrophy) and weakness
leading to loss of movement. This may affect crawling and walking
ability, arm, hand, head and neck movement, breathing and
swallowing. There are different forms of SMA and a wide spectrum of
how severely children and adults are affected.
We live in the UK and there is a treatment available called Spinraza,
which increases survival motor neuron (SMN) protein production. We are
extremely grateful to have this treatment available to us, as only a
few years ago, babies with SMA did not have this hope and would likely
not live to reach the age of 2.
Although we have this treatment option available, there is a new
pioneering much more advanced treatment called Zolgensma. Zolgensma is
a gene therapy medicine for treating SMA. It targets the genetic root
cause of SMA with a one-time-only dose and replaces the function of
the missing or nonworking survival motor neuron 1 (SMN1) gene with a
new, working copy of a human SMN gene. It is currently only available
in the USA and with a large price tag, £1.2m!
We are reaching out to our family, friends and the general public to
help us raise funds to get our son this life saving treatment. This
can be done by donating, sharing our story to everyone and anyone you
know, setting up fundraising ideas or events - whatever you can do
will be truly appreciated. We can’t do this without you and will be
eternally grateful for any donations or support, large or small.
Zolgensma will give Edward the best chance to live a fulfilling life.
From a Mum and Dad who love their son more than anything in life, we
thank you from the bottom of our hearts.