Hello, I'm Gael Mathías Puentes. I live in Colombia and I'm just 21 months old.

I was diagnosed with SMA (Spinal Muscular Atrophy), a disease that affects my muscles and my hability to move like other children. Despite everything, I'm a child full of love and zest for life.

I understand everything that's happening around me, and even though my little body doesn't respond the way I'd like, my heart and mind are full of dreams. I dream of the day I can sit up alone, eat without help, run, and play like any other child.

For that dream to come true, I need the most expensive medication in the world: Zolgensma. But this medication isn't available where I live, so my parents are doing everything they can to get it, but we need your help.

Any contribution, no matter how small, can bring me closer to that opportunity to live fully, to move, to explore the world for myself.

Is there a cure?

Currently, Zolgensma gene therapy is the most advanced option for treating SMA type 1 in children under two years of age. This single-dose treatment replaces the defective SMN1 gene, helping to restore muscle function. This treatment significantly improves life expectancy and quality of life.

In which countries is it available and how much does it cost?

Zolgensma has been approved and is available in 51 countries, with costs varying by region and agreements with governments or insurers:

United States

Canada

Japan

European Union countries (Spain, Germany, France, among others)

Brazil (government agreement for 250 babies per year)

Argentina (agreement with reimbursement based on results)

Among others

Access in Colombia

In Colombia, access to gene therapies like Zolgensma is very limited due to its high cost. It is not included in the public health system, nor are there any agreements. However, some families have turned to fundraising campaigns to pay for treatment abroad.

Conclusion

Zolgensma represents hope for children with SMA, but its high cost remains a barrier in many countries, including Colombia. For this reason, I want you to help me ensure that my dream of living is not a limitation.

If you can help us, you'd be giving me hope for a life full of possibilities.