Drug Development and Regulatory Approval

Andrew Mulberg, M.D., representing the FDA, discusses the process to get approval for a rare disease clinical trial. This is especially of interest to the families of the NNPDF as we currently have TWO clinical trials taking place for Niemann-Pick Disease. 
     1. Acid Sphingomyelinase Deficiancy (ASMD) ~ Enzyme Replacement Therapy
          Sponsor:  Genzyme, a Sanofi company, Mount Sinai School of Medicine
          To learn more about this trial ~ follow this link.  
      2.  NIH/TRND Hydroxypropyl-β-cyclodextrin (HP-β-CD) for NPC
           Sponsor:  National Institutes of Health
           To learn more about this trial ~ follow this link.
Dr. Andrew Mulberg's August 2013 presentation and professional information is noted below:
View Dr. Mulberg's Video Presentation
View Presentation Powerpoint
View Dr. Mulberg's Biography

Dr. John McKew's presentation on Therapeutics for Rare and Neglected Diseases (TRND).  Dr. McKew was one part of a series of mini-presentations referring to elements of the FDA presentation highlighted above.

Dr. John Mckew's August 2013 presentation and professional information is noted below:

View Dr. McKew's Video Presentation
View Presentation Powerpoint
View Dr. McKew's Biography

Dr. Forbes "Denny" Porter's presentation on The Cyclodextrin Phase 1 Trial.  Dr. Porter was one part of a series of mini-presentations referring to elements of the FDA presentation highlighted above.

Dr. Porter's August 2013 presentation and professional information is noted below:

View Dr. Porter's Video Presentation
View Dr. Porter's Biography

For more information on the research and these presentations see the NNPDF website at www.NNPDF.org