Spinal muscular atrophy (SMA) is a genetic condition that primarily affects muscle control. There are four types of SMA, which differ from each other in severity and age of onset. The primary symptoms of SMA are muscle weakness and motor difficulties, while severe forms can cause difficulty breathing and swallowing. The disease affects approximately 1 in 8,000 live births but currently has no cure. Research for treatment options is ongoing for this life-threatening disease.
Duchenne muscular dystrophy (DMD) is an X-linked condition that causes wasting of the skeletal and cardiac muscles. It is estimated that about 1 in 4,000 newborn males worldwide are born with this disease. The symptoms are present in early childhood and tend to progress quickly, with few individuals surviving past 30 years. There is presently no cure for DMD, though some drugs can help improve quality of life.
Cystic fibrosis (CF) is a genetic disorder occurring in about 1 in 3,000 white newborns in the United States. The disease affects many organs in the body, where buildup of thick mucus can cause breathing difficulty, digestive problems, and frequent bacterial infections. Drugs are available to manage some of these symptoms, but patients have drastically shortened lifespans. Extensive research in CF treatments is promising, with many therapies in clinical trials.