Saturday, July 22, 2017

Rare Disease Day 2012 – Spread This Call To Action To Make A Difference!

January 15, 2012 by  
Filed under Featured Stories

World Rare Disease Day 2012 is coming up on February 29th.  Consider these staggering statistics. Each year, 139 BILLION U.S. dollars are invested in medical research — much of it from tax payers Each year, 800,000 papers are published in medical journals by scientists Each year on average, ONLY 21 new drugs are approved by [...]

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

From The Valley of Death to The Valley of Cures – NIH TRND Program Supports Multiple Rare Diseases

November 15, 2011 by  
Filed under Featured Stories

Thank you Dr. Francis Collins, Dr. Chris Austin, Dr. John McKew and others at the NIH who are all involved in driving the National Institutes of Health’s Therapeutics for Rare and Neglected Diseases (TRND) program forward. TRND has been a huge success since launching five pilot projects last year, including one program that focuses on [...]

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

Moms Receive EU Orphan Drug Designation From European Medicines Agency For Novel Cholesterol Drug Called Cyclodextrin

September 14, 2011 by  
Filed under Featured Stories

Last year, after Dr. Caroline Hastings and I received the U.S. Food and Drug Administration orphan drug designation for Hydroxy-propyl-beta-cyclodextrin (HPBCD) for the treatment of Niemann Pick Type C disease, I was contacted by Sue French. Sue lives in the United Kingdom and her son William is also afflicted with the rare and fatal genetic [...]

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

FDA Filing Made Requesting Use of Medtronic SynchroMed Pump To Deliver Cyclodextrin To Brain

April 13, 2011 by  
Filed under Featured Stories

To say I am pumped up  today is an understatement!  We’ve reached another historic milestone in our effort to treat Addi and Cassi with cyclodextrin!  Dr. Caroline Hastings at Children’s Hospital Research Center Oakland filed a 400+ page document with the FDA yesterday on behalf of the twins seeking a permanent solution to deliver hydroxyl-propyl-beta-cyclodextrin [...]

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

Insulin Therapy and Alzheimer’s – Could Cyclodextrin Sugar Act on Insulin and Glucose Metabolism?

April 10, 2011 by  
Filed under Uncategorized

Recently reports in the news shows how insulin therapy may be a treatment for Alzheimer’s.  Basically, a low dose of insulin has been found to suppress the expression in the blood of four precursor proteins involved in the pathogenesis of Alzheimer’s disease, according to research by University at Buffalo endocrinologists.  Paresh Dandona, MD, PhD, University [...]

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