If you have never heard of AADC deficiency count yourself lucky. It’s a rare, incurable condition that affects only around 135 children worldwide but it’s impact on those children and their families is devastating. The children can’t speak, can’t feed themselves or hold up their head, they have severe mood swings and often suffer from … Continue reading Gene therapy is life-changing for children with a life-threatening brain disorder
AADC deficiency
How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
Logan Lacy, a child with AADC Deficiency: Photo courtesy Chambersburg Public Opinion Tomorrow, the last day in February, is Rare Disease Day. It’s a day dedicated to raising awareness about rare diseases and the impact they have on patients and their families. But the truth is rare diseases are not so rare. There are around … Continue reading How developing a treatment for a rare disease could lead to therapies for other, not-so-rare conditions
The Stem Cellar 