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Hi,
I’m raising funds for my niece in Pakistan.
Rahima, is a bright, beautiful one-year-old baby with the sweetest smile. But behind that smile, she’s fighting a rare and life-threatening disease called Spinal Muscular Atrophy (SMA).
What is SMA?
SMA is a genetic condition that affects the motor nerve cells in the spinal cord. Over time, these cells stop sending signals to the muscles, leading to muscle weakness and wasting.
- Babies with SMA often struggle to sit, crawl, or even breathe on their own.
- Without treatment, SMA can be fatal — especially in the most severe form, Type 1, which often affects infants before they reach their second birthday.
Current Treatment
Right now, Rahima is receiving risdiplam— a medication that helps her body produce more of the protein she’s missing.
- This treatment is lifelong and extremely expensive in Pakistan, costing around PKR 1.3 million (USD ~$4,500) every month.
- While risdiplam slows down the disease, it does not fix the underlying genetic cause. If we stop treatment, her condition will quickly worsen.
The Ultimate Goal — Zolgensma
The only treatment that can address the root cause of SMA is Zolgensma — a groundbreaking one-time gene therapy that replaces the faulty gene with a healthy copy.
- Cost: USD $2.1 million (over PKR 700 million) for a single infusion.
- Zolgensma offers the chance for a normal life without lifelong injections or medicines, but it must be given as early as possible to prevent further muscle loss.
Why We Need Your Help
We are in a race against time. Every day matters.
- Every penny raised will go towards keeping her alive — whether that’s continuing risdiplam to maintain her strength or moving toward Zolgensma for a permanent solution.
- Even the smallest donation can help us buy more time and hope.
Raising millions might seem an impossible challenge but together we can move mountains and save a child’s life! The biggest fundraiser on Gofundme: $45 million - Americas Food Fund! All thanks to people like you. 2.1 million is only 5% of that immense achievement. We’re starting small with £50,000 but hope to build momentum and reach our ultimate goal with providing Rahima the Zolgensma gene therapy.
From the bottom of our hearts, thank you for giving Rahima a fighting chance. Together, we can give her the gift of life.
Organizer
Tayyaba Qureshi
Organizer