Disease Area
kennedy's Disease
Spinal and bulbar muscular atrophy (SBMA)
Typical symptoms are muscle weakness, atrophy, and bulbar muscle dysfunction. Bulbar weakness causes difficulty with speech and swallowing, which can lead to aspiration pneumonia. Patients expect normal lifespan, but with long-term wheelchair dependence.
🧠 1 in 7,000 Males Affected in the U.S. and Europe
Only males are affected, and 1 in 40,000 males are diagnosed.
⏳ Ages 30–50
The most common window for symptom onset, striking during peak working and family-raising years
🚫 0 Disease-Modifying Therapies
No approved treatments currently exist to slow, stop, or reverse disease progression
💸 Annually $350K / Patient
Annual care costs driven by medical visits, long-term medications, physical and psychiatric care, assistive devices, and increasing caregiving needs as independence is lost
🧬 X-Linked Inheritance Risk
Affected male passes mutated X-chromosomes to all his daughters. Children have a 50% chance of inheriting the SBMA mutation from a carrier female, extending the impact far beyond those already diagnosed.
Our Mission & Vision
Our mission is to develop scalable, small-molecule therapies that directly target the RNA-level cause of Kennedy's disease and enable safe, long-term treatment for patients.
