08/18/2026
This , join us in advocating for expanded newborn screening, equitable access to emerging therapies, and sustainable care for every rare family.
Here is what you need to know about SMA and why early intervention changes everything:
🧬 Cause:
is caused by a mutation or deletion in the SMN1 gene. Without a functional gene, motor neurons progressively deteriorate, impacting a child’s ability to walk, eat, and breathe
👶 Prevalence:
Approximately 1 in 11,000 live births worldwide are affected by SMA
🧾 Carrier Rate:
About 1 in 50 people carry the mutated gene, usually without any family history or symptoms
📊 Severity:
SMA is classified into four main types , where Type 1 is the most severe and accounts for roughly 60% of diagnoses. Untreated infants with Type 1 rarely survive past age two
💊 Life-Changing Therapies:
While there is no universal cure, groundbreaking gene therapy including Spinraza®, Zolgensma®, Evrysdi®, and Itvisma®—have transformed outcomes, especially when administered before symptoms appear
🔬 The Diagnostic Race:
Diagnostic delays remain a major hurdle worldwide. When newborn screening or early genetic testing is delayed, motor neuron loss becomes irreversible before treatment even begins
In Eastern Europe and Ukraine, navigating an SMA diagnosis presents unique challenges. Our BfS SMA Program focuses where time and resources are most critical:
🫁 Funding essential breathing equipment to protect respiratory health
🏥 Covering time-sensitive surgeries to prevent severe spinal complications
🏋️♂️ Providing annual rehabilitation courses to preserve motor function
No parent should have to fight this battle alone—and no child’s survival should depend on their geographic location 🇺🇦🇺🇸🕊️
Learn more about our SMA Program and stand with our families:
🧬 https://birdsforsofia.org
(link is also in our bio)
Thank you!
🕊♥️
