Thursday, January 27, 2011
Update from Genzyme on Acid Sphingomyelinase Deficiency (ASMD) Clinical Trials
A note from Genzyme - January 2011:
Genzyme is continuing efforts to develop recombinant human acid sphingomyelinase (rhASM) for the potential treatment of the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease Types A and B). After completing the Phase 1 clinical trial in 2009, we engaged regulatory authorities in discussion about plans for a Phase 2 clinical trial and conducted additional preclinical research in 2010. This regulatory dialogue is ongoing. We remain committed to the development of a therapy for ASMD and will keep the community informed once our regulatory discussions are complete and we can confirm a start date for the Phase 2 clinical trial for Niemann-Pick B patients.
We are pleased to note that an abstract from the Phase 1 clinical trial was selected for a podium presentation by Dr. Margaret McGovern at the 7th annual lysosomal disease network's WORLD conference, to be held in Las Vegas, February 16-18, 2011.
Genzyme Corporation
500 Kendall Street
Cambridge, MA 02142
www.genzyme.com
(To learn more about Niemann-Pick Disease, visit www.nnpdf.org. To learn more about these clinical trials, please visit the National Niemann-Pick Disease Foundation's Enzyme Replacement Therapy page.)
Labels:
ASMD,
enzyme,
Genzyme,
niemann-pick disease,
NNPDF,
replacement,
therapy,
Type A/B
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