Summary

Providing 15 children in Karakalpakstan with a diagnosis of Duchenne Myopathy (a rare genetic disease associated with muscle weakness or dysfunction. The child's muscles of movement and breathing gradually begin to deteriorate) with a Verticalizer (a special wheelchair for the child to move in an upright position) and IVL (Artificial Respiratory Equipment). This way, we will ensure that the children can study, move, play, and breathe freely, just like other children.

Challenge

Today, there are more than 1,000 children in Uzbekistan diagnosed with Duchenne Myopathy. This diagnosis is a rare genetic disease associated with muscle weakness or failure, which gradually damages the child's muscles of movement and breathing. There is no cure for this disease in Uzbekistan. Abroad, 5 types of drugs can treat this disease: translarna (ataluren), viltepso (viltolarsen), viondis 53 (holodirsen), amondis (casimersen), exondis 51 (eteplirsen). However, these drugs require at least 3 years of treatment. This can cost from $100,000 to $2 million per child.

Solution

Right now, more than 1,000 children in Uzbekistan are living with Duchenne Myopathy, a rare genetic disease that progressively weakens their muscles, including those needed for movement and breathing. Without a cure available in Uzbekistan, life-saving medications used abroad remain out of reach for many due to their staggering costs, ranging from $100,000 to $2 million over a three-year treatment period.

But while we wait for affordable treatment options to become a reality, we can act now to improve their quality of life. These children urgently need two essential devices:

• Verticalizers – Special wheelchairs that allow them to stand and move vertically, enabling mobility and independence.
• Ventilators – Artificial respiration devices that support their ability to breathe freely.

These tools can make an immediate difference, allowing children with Duchenne Myopathy to study, play, move, and enjoy life more fully, just like other children.

You can help bring hope to these children and their families. Your support can provide them with the critical equipment they need to live more comfortable and fulfilling lives.

Let’s come together to ease their burden and give them the chance to thrive. Donate today and make a lasting impact!