Support Hurain's Piggy Bank to Help Her Fight Against SMA

Introduction:

I'm Waseem Ahmed, originally from Pakistan and I'm reaching out to you with a plea from the depth of my heart. My daughter, Hurain Waseem (who is 2.7 years old, weighing only 9.5kg), is in a fierce battle against Spinal Muscular Atrophy (SMA), a cruel condition that's stealing her ability to move day by day.

Background:

Hurain, our one and only treasured daughter was granted to us after years of hopeful waiting, but SMA confines her fragile body, denying her the chance to pursue her dreams. Hurain's journey with SMA has been heartbreaking and extremely emotional for us. At the earlier stage, up to 10 months, she showed no signs of this relentless disease. Till that time, she was able to move her hands and sit on her own for a few minutes like every other regular child at her age. Our first alarming incident was when we were trying to stand her up by keeping her legs straight as per the doctor’s advice, but her hands and feet started getting sweaty and red. This was shocking as the late walkers never showed these kinds of signs. At that moment our mind was telling us that something was not right as it could be a Neuro problem. We decided to take her to a Neurologist and that visit turned into a nightmare when the diagnosis revealed that she was born with SMA. And the worst nightmare was when the Doctor told us that there is no cure for SMA available. Our little world shattered, all we needed to know was if and how she would survive?

Journey Towards Hope:

As time passed, she grew weaker, losing her grip and her ability to keep her head straight up. At this moment, we decided not to give up on her. We started researching to get to know more about this disease and we've learned about a permanent solution in the form of “Zolgensma”, a medication used for the treatment of SMA through which our little angel would finally stand up on her feet. However, the happiness lasted for a few instants as we discovered that the cost for this treatment is a staggering $2.1 million, which is way beyond our means.

Reference (cost): https://www.theguardian.com/science/2019/may/25/21m-novartis-gene-therapy-to-become-worlds-most-expensive-drug#:~:text=Swiss%20drugmaker%20Novartis%20has%20received,those%20not%20yet%20showing%20symptoms

Treatment:

Previously, this treatment wasn't accessible for children above 2 years of age. However, the latest clinical trial has extended the eligibility for Zolgensma to children weighing up to 21kg. Hurain is just about 9.5kgs (since last year) even at this age. The weakness comes with this condition, according to the doctors. Now, we're in a race against time to save her.

Reference (Clinical trial results): https://clinicaltrials.gov/study/NCT04851873?tab=results

Case Files:

https://drive.google.com/file/d/1zaVfGgq-DrVbk7SGiPEf1JyYyzwOWLH6/view?usp=sharing

Fund Allocation:

To ensure your contributions make a direct impact, all funds will go straight to Novartis Pharmaceuticals (the manufacturer of Zolgensma) or the authorized medical facility. We believe in complete transparency and will keep our supporters informed with regular updates on how their donations are helping Hurain on her journey.

Any funds not used for services related to the administration of Zolgnemsa will be donated in full to the United States non-profit corporation named TRANSPARENT HANDS FOUNDATION, INC, based in Arlington, VA.

Every contribution, big or small, will make a huge difference in Hurain's life giving her the chance she deserves to live a happy and healthy life.

Your kindness and generosity could make all the difference in the world, just as it can give a little girl her strength back…