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Nonprofit Group Works to Raise Rare Disease Awareness in India

With an estimated 1.37 billion inhabitants, India will likely surpass China in five years as the world’s most populous country. That also means it will have more rare-disease patients than any nation. It already has more than twice as many as the 28-member European Union. Harsha K. Rajasimha, a genomics…

Starting Kalydeco Treatment Before Birth May Help Halt CF Progression Post-Birth, Animal Study Suggests

Treating an animal model of cystic fibrosis (CF) with Kalydeco (ivacaftor) during embryonic development and after birth revealed that early treatment improved pancreatic function, delayed lung disease, and reduced infections, a study suggests. The study, titled “In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model…