ASMD (Niemann-Pick Disease Type A/B & B) Updates from the 2015 WORLD Symposium

Dear NNPDF Families and Friends,

The National Niemann-Pick Disease Foundation is excited to share with our NPD community family membership research updates presented at the 2015 WORLD Symposium on Lysosomal Disease Research under the direction of: Dr. Melissa Wasserstein M.D. & Dr. Beth Thurberg, MD, PhD, of Genzyme, a Sanofi Company. This research is in support of Genzyme’s ongoing efforts to develop a potential therapy for acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease Types A and B).

Visit the Enzyme Replacement Therapy page to view:

- Genzyme Press Release ~ Dateline 02/12/2015

- Related video interview with Dr. Beth Thurnberg

- Dr. Wasserstein & Dr. Thurnberg's presentation abstracts

The NNPDF and all of the members of our ASMD family community wish to extend a HUGE note of thanks to the 5 Phase 1B clinical trial participants in and taking on this brave effort which involved extreme commitment of time, travel, work and family involvement in support of the wider ASMD (NPD Type B) community and patients worldwide.


We will continue to bring you the latest research from the Symposium floor as it is made available to us. In the meantime, you can read all the abstract research on the NNPDF WORLD Symposium web page: http://www.nnpdf.org/WORLDSymposium.html