b78aae812502f001d40a869e557ce154.jpg

SMA Australia

SMA AUSTRALIA

Phone: (03) 9796 5744

exp fillerMY SPINRAZA JOURNEY

My name is Ismail Gul and I was born with Spinal Muscular Atrophy Type 1 which is the most severe form of the disease. I am currently 36 years old and require 24/7 one to one intensive care from my mother. Despite my severe disability, I still try to live as normal a life as possible. I’m a very social person and am well known throughout my local community. Some of my hobbies include, reading, watching movies, playing video games and going out when the weather is nice.

Around 6 years ago, I came across some information online which stated that treatments were being developed for SMA. This sparked a high level of interest from me and I began researching night and day, not only into the upcoming treatments, but also the science behind SMA itself. I was in constant contact with professors and researchers, both from Australia and overseas as well. After my years of research, I was eventually able to join the Biogen Early Access Program for Spinraza thanks to professor Monique Ryan and the wonderful people at the Royal Children's Hospital.

It was both a dream come true and scary at the same time. I was heading into unknown territory, not knowing what to expect.

Before starting on Spinraza, I used to have an extremely difficult time going to the toilet (urinating). It had gotten worse over time until I reached the point where it would take me anywhere from 10 minutes to half an hour and would even require multiple attempts. I was on the verge of having to use a catheter and this is something that I never wanted to do and is one of my worst fears.

After being on Spinraza for about 5 weeks, my mother and I noticed that it was becoming easier for me to go to the toilet. This had a profound impact on my quality of life. As time went by, it became easier and easier until I could do it normally again. This made things much easier, both at home and when out and about. I was no longer in fear of having to have a catheter and I also felt more like a normal person as well.

This benefit may not sound like much to some however it means a lot to me as it has had a major impact on my quality of life. I cannot state this enough.

Another benefit that I have seen since using Spinraza is that I have regained some slight movement in my left thumb which I had lost about 8 years ago. This has made it much easier for me to use my computer and to access more devices.

Currently these are the only two benefits we have seen however they are very impactful to my quality of life. I am hoping that with subsequent dosing, to see further benefits in the future.

Lastly, I would like to thank everyone involved for making all of this possible and I would also like to take this opportunity to thank my mother for looking after me all of these years.

Thank You

Ismail Gul