International Niemann–Pick Disease Alliance
For six years, The Wall Street Journal followed a group of parents and scientists seeking a treatment for a rare and fatal genetic disease that strikes primarily children. Their collaboration accelerated development of a promising drug and, along the...
An article highlighting the INPDA’s campaign to improve diagnosis of NP-C is featured in today’s BBC News. Article can be viewed here: http://m.bbc.co.uk/news/health-31644877