The mission of Birds For Sofia is to support children diagnosed with rare diseases from the underrepresented parts of the world. We help families to get access to gene therapy. We fast-forward the fundraising process before it is too late.

One of the major rare diseases we support is spinal muscular atrophy (SMA). It is a fatal genetic disease. SMA affects approximately one in every ten thousand babies born each year. Most of them do not live past two years of age.

One of the leading medicines to treat SMA, Zolgensma (by Novartis), is recognized by the Guinness Book of World Records as the most expensive medicine in the world ($2.1 million per one injection). Zolgensma replaces the function of the missing or non-working SMN1 gene with a new working copy of the human SMN1gene. In essence, it halts the disease at its root.

Zolgensma's accessibility is extremely low due to its astronomically high cost. Today, in many parts of the world, hundreds of SMA-afflicted children cannot receive the gene-based therapy they desperately need to survive.

https://birdsforsofia.org/