Scientists use drug to repair rare birth defect

Scientists have developed a potential cure for a rare eye disease, showing for the first time that a drug can repair a birth defect. They formulated the drug Ataluren into eye drops, and found that it consistently restored normal vision in mice who had aniridia, a condition that severely limits the vision of about 5,000 […]

Rare cause of anemia in newborns often overlooked, research suggests

Some babies diagnosed with and treated for a bone marrow failure disorder, called Diamond Blackfan Anemia, may actually be affected by a very rare anemia syndrome that has a different disease course and treatment, say scientists. ScienceDaily: Infant Health News