The National Niemann-Pick Disease Foundation supports and promotes research
to find treatments and a cure for all types of Niemann-Pick Disease, and
we provide support services for individuals and families affected by NPD. Our
Vision...is a world where Niemann-Pick Disease is no longer a threat
to a full and productive life for patients and their families..
Genzyme ERT Clinical Trial Update
JUNE 27th, 2014
Left to right, Alan Gilstrap, Director, Advocacy Development - Rare Disease; Genzyme Corporation ; Sandra Cowie, NNPDF Research Committee Chairperson ; Dr. Simon Jones, Manchester Centre for Genomic Medicine St. Mary's Hospital, University of Manchester, England; Nadine Hill, NNPDF Executive Director
For the Updates
NIH Cyclodextrin Trial
~ UPDATE ~
JUNE 27th, 2014
We received the following update from Dr. Denny Porter with reference to the NIH/TRND Cyclodextrin Clinical
Research News & Announcements:
Family Resources & Support:
“The National Niemann-Pick Disease Foundation (NNPDF) does not engage
in the practice of medicine. It is not a medical authority nor does it
claim to have medical knowledge. This site is an educational service
of the National Niemann-Pick Disease Foundation and is not meant to provide
diagnostic or treatment advice. Information contained or suggested on
this Web site does not constitute medical advice. For all information
related to care, medication or treatment, the NNPDF recommends consulting
a physician to determine if information presented is applicable. Please
review these additional cautions about medical information
provided on the Internet.?
This Web site is a service of the National Niemann-Pick Disease Foundation's Family Services Program, made possible by the financial support of our member families and other generous donors and grantors. This site receives no government funding, nor does it host or receive funding from advertising or from the display of commercial content.
This site complies to the HONcode
standard for trustworthy health information:
The National Niemann-Pick Disease Foundation is a 501(c)(3) organization.
Your donations are fully tax-deductible.