Category Archives: NPCD Research Updates

$50,000 CODRT Grant

Our scientists have been rewarded a grant for further research…. we’re getting closer and closer as each dollar gets committed to NPCD research. Million Dollar Bike Ride Grant Program from Orphan Disease Center, University of Pennsylvania Principal Investigator: Professor Ashley Bush Project title: Dual targeting of defective lipid and metal pathways in Niemann-Pick type C disease:…Continue Reading

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.…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

Zavesca/Miglustat update from US FDA Panel shows some hope for Australia

UPDATE 2-US FDA panel backs new use for Actelion drug Tue Jan 12, 2010 5:31pm Stocks Regulatory News Healthcare * FDA decision due by March 10 * Agency usually follows panel recommendations (Adds panelist, company comments) By Lisa Richwine SILVER SPRING, Md., Jan 12 (Reuters) – A U.S. advisory panel on Tuesday recommended approval of…Continue Reading

Dr. Marc Patterson a paediatric neurologist at Mayo Clinic discusses Niemann-Pick disease type C.

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