International Niemann–Pick Disease Alliance
Genzyme, a Sanofi company, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olipudase alfa. This enzyme replacement therapy is being investigated for the treatment of patients with nonneurological manifestations of acid sphingomyelinase...
Ebola’s ‘Achilles heel’ has been identified by a team of scientists, prompting hopes of new treatments to stop the virus spreading. Researchers in New York believe they have located the vital protein, which affects whether or not the virus...
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