Sunday, October 22, 2017

FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease

May 16, 2010 by  
Filed under Featured Stories

Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail (probably next week), it appears our Orphan [...]

Share this:
  • Print
  • Digg
  • del.icio.us
  • Facebook
  • Google Bookmarks
  • Diigo
  • email
  • PDF
  • Twitter
  • Yahoo! Bookmarks

FDA Does Not Approve Actelion’s Zavesca in US – Distressing News For Niemann Pick Type C Community

March 9, 2010 by  
Filed under Featured Stories

Actelion, the makers of Zavesca, today put out a brief statement saying that the U.S. Food and Drug Administration did not approve Zavesca and wants more information from the company on treating Niemann Pick Type C, a rare and dementing neurological disease that afflicts our six year old identical twins, Addi and Cassi. Niemann Pick Type C disease is caused [...]

Share this:
  • Print
  • Digg
  • del.icio.us
  • Facebook
  • Google Bookmarks
  • Diigo
  • email
  • PDF
  • Twitter
  • Yahoo! Bookmarks

FDA Orphan Drug Workshop Makes Filing Applications Easy – Even For Rare Disease Patient Advocates!

February 27, 2010 by  
Filed under Featured Stories

At a historic orphan drug workshop held by the U.S. Food and Drug Administration Feb. 25-26 and before World Rare Disease Day 2010, we filed a momentous orphan drug application with the FDA for the treatment of Niemann Pick Type C disease with cyclodextrin. Our filing occurred in Claremont, CA, at the Keck Graduate Institute’s Center [...]

Share this:
  • Print
  • Digg
  • del.icio.us
  • Facebook
  • Google Bookmarks
  • Diigo
  • email
  • PDF
  • Twitter
  • Yahoo! Bookmarks

Cystic Fibrosis and Niemann Pick Type C Linked Through Cholesterol Abnormalities and cAMP Cell Signaling Pathway

April 26, 2009 by  
Filed under Cystic Fibrosis, Related Diseases

A few months ago, I wrote a blog about the connection points between Duchenne Muscular Dystrophy (DMD) and Niemann Pick Type C (NPC) and how more research dollars are needed for collaborative research projects between the two deadly diseases. Today, I am writing about the connections between Cystic Fibrosis (CF) and Niemann Pick Type C. [...]

Share this:
  • Print
  • Digg
  • del.icio.us
  • Facebook
  • Google Bookmarks
  • Diigo
  • email
  • PDF
  • Twitter
  • Yahoo! Bookmarks