Thursday, April 26, 2012

Update from Genzyme on ASMD Clinical Trials

Update on Clinical Trial of Enzyme Replacement Therapy (ERT) for Acid Sphingomyelinase Deficiency (ASMD)

The National Niemann-Pick Disease Foundation (NNPDF) has been staying in touch with representatives from Genzyme with regard to the status of Phase 2 of the Enzyme Replacement Therapy (ERT) clinical trial for Acid Sphingomyelinase Deficiency (ASMD) NPD Type B.

It seems there has been some confusion among members of the ASMD community; please note that we have contacted Genzyme and confirmed that the Phase 2 trial has not been cancelled and the Genzyme/Sanofi Company is committed to the ongoing support of our NPD Type A and B patients and families.

Genzyme has advised that they are still actively preparing for a Phase 2 clinical trial for enzyme replacement therapy in ASMD/NPD Type B. The trial is expected to evaluate the safety and efficacy of different doses of rhASM when administered once every two weeks.

The program remains a key priority for Sanofi. They are committed to the Niemann-Pick community and have provided the NNPDF with an update on the Genzyme-sponsored Acid Sphingomyelinase Deficiency (ASMD) Clinical Trials.

To read the update from Genzyme, visit the NNPDF's NewsLine or Enzyme Replacement Therapy Type B page.  To learn more about Niemann-Pick Disease of all types, visit

Thursday, April 19, 2012

Joshua Jaylon Garcia

Joshua Jaylon Garcia
2/2/90 - 4/13/12

With great sadness, we pass along word of yet another young life lost to Niemann-Pick Disease Type C.

Joshua Jaylon Garcia, the son of Michelle Pino of San Felipe Pueblo, NM, and Tony Garcia of Albuquerque, NM, lost his battle with NPC on April 13, at the age of 22 years.

In addition to his parents, Joshua leaves behind a younger sister, Alicia, grandparents, aunts and uncles, cousins, and other relatives and friends.

Our deepest sympathy to Joshua's family and friends in their loss.

To learn more about Niemann-Pick Disease, visit

Friday, April 13, 2012

James "Wilson" Speakmon-Coleman

James "Wilson"
4/8/93 - 4/1/12

We are very sad to report that James "Wilson" Speakmon-Coleman has passed away from the effects of Niemann-Pick Disease Type C (NPC). The son of David P. Coleman and Theresa Speakmon-Coleman, both of Portland, Oregon, Wilson was one week short of his 19th birthday. In addition to his parents, Wilson is survived by his younger brother, Thomas, grandparents, and other relatives and friends.

Services were held April 10, at Mt. Scott Funeral Home, Portland. The family has requested that memorials be made to the NNPDF in Wilson's memory.

Our deepest sympathies to the Speakmon-Coleman family at this very sad time.

For more information about Niemann-Pick Disease, visit