The team at Aceragen is working rapidly with our lead program ACG-801 to initiate a clinical trial for investigational enzyme replacement therapy treatment of acid ceramidase deficiency presenting as Farber disease. We also plan to develop treatments for additional rare diseases with high unmet need.
Farber disease is a rare metabolic disease that usually has four hallmark symptoms that appear in very early childhood.
- Joint pain or contractures
- Painful and swollen joints
- Bumps or nodules under the skin
- Increasing weak or hoarse voice
These symptoms can appear at any age and any order. They can also vary in severity and how quickly they progress, making it hard to diagnose. Most patients are initially misdiagnosed as having juvenile idiopathic arthritis