Dossier
maple syrup urine disease
Coverage of maple syrup urine disease in the Nexus archive.
- 2 Philadelphia labs receive millions in federal funding to create treatments for rare genetic diseases in 5 years
Two Philadelphia-based labs received federal funding to develop gene therapies for rare genetic diseases, aiming for human trials in three years. The funding supports research at Children’s Hospital of Philadelphia and GEMMABio, focusing on scalable treatments for Maple syrup urine disease and homozygous familial hypercholesterolemia.
- What is ‘maple syrup urine disease’ — and when can it turn fatal?
Maple syrup urine disease (MSUD) is a health condition that can cause unusually smelly urine and is potentially life-threatening.