Help Ava Walk Again!
Donation protected
Meet Ava - born on a Friday 13th - she made her parents the luckiest mom and dad in the world. Everything about Ava was perfect - including her beautiful eyes and cheeky smile. However at 18 months old, while other kids were playing, running, and climbing, Ava struggled to crawl, sit up, bend down or stand up without support. It seemed like everyday, Ava was getting weaker. Ava's parents went on a year long mission, going from doctor to doctor, before she was finally diagnosed with Spinal Muscular Atrophy (SMA).
SMA is a rare genetic disorder and is the number 1 cause of infant deaths. It is characterized by the absence of a gene that produces a protein called SMN1 that feeds motor neurons. When not enough protein is produced, the neurons don't develop and eventually die, causing weakness and atrophy of the muscles. This results not only in the inability to walk, but breathing, swallowing and digestion are affected.
SMA is divided into 4 categories, type 1,2,3 and 4. These are general guides, and can overlap each other. Type 1, the most severe, causes death by the age of two, as muscles are not strong enough to perform for breathing. Type 2, are not able to walk, have to use assisted breathing at night, and some use feeding tubes.
Ava is considered a "weak type 3" as she is closer to type 2. She started standing around 2 years old, and took a few steps with assistance. However, she was never able to crawl up the stairs, or go from sitting to standing. She's even unable to sit-up while lying down. In March of last year, she fell and fractured her leg. Since then, she has not been able to bear weight again (stand on her own), even with extensive physiotherapy, aqua therapy, stretching etc. She is currently in a wheelchair full time.
Without treatment, Ava will likely need breathing and cough assisting machines, and possibly a feeding tube. So even though she is a type 3, she may eventually present as a type 2. One of the main risks for SMA patients is that they are very susceptible to lung and breathing complications, which in some cases can be fatal. Ava's parents fear her choking on her phlegm or getting pneumonia every time she gets a simple cold.
Is there a cure?
No, there isn't.
But there is hope.
On June 30, 2017, Health Canada approved the first drug that would treat SMA. This drug has been developed for over ten years and clinical trials have shown significant improvement. One would say rather miraculous, as type 1 patients survived, some were able to sit up unassisted, and some even walked for the first time! The drug has shown not only to stop but also reverse the damage and symptoms, and the earlier it is given, the more effective it is. Infants born with SMA were given the treatment at birth and grew up showing no symptoms!
How much does Spinraza cost?
Though Health Canada has approved the drug, Spinraza, the provinces have not approved funding, yet. Spinraza's maker, Biogen, has given parents the opportunity to purchase the drug at a discounted cost of approximately $63,000 per dose. 6 doses are needed in the first year and 3 every year after that.
Ava's parents are lucky though - their insurance has agreed to cover some (but not all) of the costs to fund the medicine for Ava. The first 4 doses have (so far) cost Ava's parents $46,000. The 5th dose unfortunately will not be covered by insurance at all, due to timing. That's over $100,000 in medical expenses so far, with treatment required for life!
How do we know it's going to work?
Ava's parents know this medicine is worth it and working! Since receiving the first four doses, Ava has already improved!! She has been able to walk up to 18 meters in her walker compared to only 5 meters before her first dose! She is definitely getting stronger with her "muscle medicine" and her parents are positive with time, she will be back on her feet, standing and walking! And let's be honest - stopping the deterioration is just as important as gaining strength!
Please help Ava's parents purchase the 5th dose of medicine for their sweet princess and give her muscles a chance of surviving and growing stronger. Help Ava to walk again!!!
Thank you all for your donations! For more information on Spinal Muscular Atrophy, please check out www.curesma.ca or www.curesma.org
SMA is a rare genetic disorder and is the number 1 cause of infant deaths. It is characterized by the absence of a gene that produces a protein called SMN1 that feeds motor neurons. When not enough protein is produced, the neurons don't develop and eventually die, causing weakness and atrophy of the muscles. This results not only in the inability to walk, but breathing, swallowing and digestion are affected.
SMA is divided into 4 categories, type 1,2,3 and 4. These are general guides, and can overlap each other. Type 1, the most severe, causes death by the age of two, as muscles are not strong enough to perform for breathing. Type 2, are not able to walk, have to use assisted breathing at night, and some use feeding tubes.
Ava is considered a "weak type 3" as she is closer to type 2. She started standing around 2 years old, and took a few steps with assistance. However, she was never able to crawl up the stairs, or go from sitting to standing. She's even unable to sit-up while lying down. In March of last year, she fell and fractured her leg. Since then, she has not been able to bear weight again (stand on her own), even with extensive physiotherapy, aqua therapy, stretching etc. She is currently in a wheelchair full time.
Without treatment, Ava will likely need breathing and cough assisting machines, and possibly a feeding tube. So even though she is a type 3, she may eventually present as a type 2. One of the main risks for SMA patients is that they are very susceptible to lung and breathing complications, which in some cases can be fatal. Ava's parents fear her choking on her phlegm or getting pneumonia every time she gets a simple cold.
Is there a cure?
No, there isn't.
But there is hope.
On June 30, 2017, Health Canada approved the first drug that would treat SMA. This drug has been developed for over ten years and clinical trials have shown significant improvement. One would say rather miraculous, as type 1 patients survived, some were able to sit up unassisted, and some even walked for the first time! The drug has shown not only to stop but also reverse the damage and symptoms, and the earlier it is given, the more effective it is. Infants born with SMA were given the treatment at birth and grew up showing no symptoms!
How much does Spinraza cost?
Though Health Canada has approved the drug, Spinraza, the provinces have not approved funding, yet. Spinraza's maker, Biogen, has given parents the opportunity to purchase the drug at a discounted cost of approximately $63,000 per dose. 6 doses are needed in the first year and 3 every year after that.
Ava's parents are lucky though - their insurance has agreed to cover some (but not all) of the costs to fund the medicine for Ava. The first 4 doses have (so far) cost Ava's parents $46,000. The 5th dose unfortunately will not be covered by insurance at all, due to timing. That's over $100,000 in medical expenses so far, with treatment required for life!
How do we know it's going to work?
Ava's parents know this medicine is worth it and working! Since receiving the first four doses, Ava has already improved!! She has been able to walk up to 18 meters in her walker compared to only 5 meters before her first dose! She is definitely getting stronger with her "muscle medicine" and her parents are positive with time, she will be back on her feet, standing and walking! And let's be honest - stopping the deterioration is just as important as gaining strength!
Please help Ava's parents purchase the 5th dose of medicine for their sweet princess and give her muscles a chance of surviving and growing stronger. Help Ava to walk again!!!
Thank you all for your donations! For more information on Spinal Muscular Atrophy, please check out www.curesma.ca or www.curesma.org
Organizer
Yasmine Hakimpour
Organizer
Mississauga, ON
