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Posted by on Feb 20, 2014 in Foundation NewsLine |

UPDATE: Genzyme ASMD ERT 1b ERT Clinical Trial

Hello NNPDF Families and Friends:

Genzyme, a Sanofi company, is pleased to update the Niemann-Pick Disease patient community on the progress of their efforts to develop a potential therapy for acid sphingomyelinase deficiency (ASMD, also known as Niemann-Pick disease Types A and B).

Dr. Simon Jones, MbChB, presented interim tolerability and safety information from the Phase 1b clinical trial at the WORLD(We’re Organizing Rare Lysosomal Diseases) Symposium, held in San Diego, CA; Tuesday, February 11th thru Thursday, February 13th, 2014.

The title of the presentation was: “An open-label, multicenter, ascending dose study of the tolerability and safety of recombinant human acid sphingomyelinase (rhASM) in patients with ASM deficiency (ASMD)” and was presented by Dr. Simon Jones.

To read the full announcement from Genzyme, A Sanofi Company; an accompanying statement from the NNPDF Research Committee Chair, Sandra Cowie and a narrative title: A Patients Perspective on ERT Therapy by James Dyson ~ please follow the link below.


The NNPDF and our ASMD family membership continue to be grateful to the team of members at Genzyme/Sanofi and the medical/clinical professionals at Mt. Sinai and UK clinical sites for their ongoing commitment and determination to move this ERT clinical trial forward. It is appreciated.

We will all anxiously await the further details highlighting the trial data results as they become available.

Kind regards,
Nadine

S 
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