Category Archives: General News

USA – UPDATE: NIH/TRND NPC Cyclodextrin Trial

Hello Friends of the NNPDF, We have received the following update from Dr. Denny Porter and Nicole Yanjanin of the NIH/TRND NPC Research Team with regards to the status on the NPC Cyclodextrin Clinical Trials. Dear NPD Community, The NIH cyclodextrin trial has started and the first patient will receive the drug on Monday 2/4. We are very excited to get started and to get data that will direct future studies of cyclodextrin in NPC. We have been in contact with many families and would like to provide an update about the trial screening process. The patients in this trial are grouped into “cohorts” of three patients each. Because we did not know exactly when we would be able to start the trial, the first three patients were selected because they live relatively close to the NIH or were able to come to the NIH with very little notice (less than 2 weeks in one case). Only the first 3 patients have been scheduled. We have not yet filled the remaining slots for the trial. The FDA asked us to start at a lower dose than we initially proposed. The initial data from these first three patients will determine what exactly we will do next. We decided that we will need to re-screen patients when we are ready to schedule the next cohort (patients 4, 5 and 6), in order to make sure they are still eligible. This will also need to happen again with the last 3 patients. We have a screening log of all individuals who we have screened and will be in touch with families when we are prepared to schedule the remaining slots in the trial. We would like to thank you for your continued patience and we apologize for any delays in returning phone calls or emails. We are doing our best to get this trial going as quickly and safely as possible so we can answer questions about cyclodextrin in NPC. Please do not hesitate to contact us with additional questions. The NIH NPC Research Team The NNPDF will continue to update our WebSite and Facebook Awareness pages with these periodic releases for future reference. Please follow the following web pages for more detailed and background information: ~ NNPDF Cyclodextrin Web page: http://www.nnpdf.org/Cyclodextrin.html ~ NNPDF Newsline Updates Web page: http://www.nnpdf.org/NewsLinePage2012.html ~ NNPDF Facebook Awareness page: http://www.facebook.com/groups/nnpdf/ Nadine M. Hill Executive Director National Niemann-Pick Disease Foundation, Inc. 401 Madison Avenue, Suite B; PO Box 49 Fort Atkinson, WI 53538-0049 Phone: 920-563-0930 Fax: 920-563-0931 E-mail: nhill@nnpdf.org Web site: www.nnpdf.org

Kirsten Smith

“Gone to meet her brother Matty in heaven”. Kirsten passed on Thursday 16th January, 2014. Aged 39 years and 11 months. Donations to foundation in lieu of flowers are appreciated by the Smith family.Continue Reading

Band Bash at Bundalong

Come along to the Bundalong Tavern (7419 Murray Valley Road, Bundalong) and enjoy live music from Melbourne’s Sheila’s of the 70′s and local talent Motnelle and Fanbelt. Entry is $20 per adult or $50 family. Bring the kids for face painting and games, sausage sizzle, trade stalls, and craft and auction selling donated goods. All monies raised…Continue Reading

FDA Approves Request For New Cyclodextrin Treatment For Niemann Pick Type C

FDA Approval Received! Children’s Hospital Oakland Receives FDA Clearance to Begin World’s First Cyclodextrin Administration Into the Brains of Twins with Rare and Deadly Cholesterol Disease Sugar Molecule Used In Common Food and Household Products Like Febreze® Fabric Refresher Called Hydroxypropyl Beta Cyclodextrin (HPßCD) Will be Delivered into Twins’ Central Nervous System in an Attempt…Continue Reading

Cycle 4 Sam – Shining his star… Part II

Matty & Timmy Lloyd chat with Marty before the start of day 2 Mandy & Paul checking out the day’s route. A quick riders briefing before the team head off Releasing 21 pink balloons in memory of Cassie Stallard RIP Paul & Mandy Jacobs lead the team through town in the Truck On their way…..…Continue Reading

Merchandise page up and running….

Hi Everyone, Just a quick note to let you all know that the Australian NPC Disease Foundation Inc website now has NPC merchandise for sale! At the moment it is only set up for Australian shoppers, however until I sort out the international side, our overseas shoppers can still order simply by emailing mandy@npcd.org.au Check…Continue Reading

Kaitlyn Maher Sings You Were Meant To Be for Dying Kids With “Childhood …

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