Clinical trials

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Clinical trials in the pipeline from around the world.
These are absolutely worth keeping an eye on!


Go to the link below.  Put Pompe into the search box.

http://www.clinicaltrials.gov/


Gene Therapy Trials

Enzyme Replacement Therapy Trials

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About Clinical Studies for Rare Diseases


​The development of an investigational treatment is divided into steps called ‘phases’. The investigational treatment generally passes through each phase successfully before it can continue on to the next.

PHASE 1  
3-50 people
  1. Usually healthy volunteers but can be people with the disease
  2. Safety and how the body breaks down the study treatment

PHASE 1/2 OR 2
10-60 people
  1. People with the disease
  2. Safety and effectiveness (the ability of a study treatment to produce a beneficial change)

PHASE 3
12-150 people
  1. People with the disease
  2. Long-term safety and effectiveness compared to current standard of care or a placebo (a sugar pill)
  3. Once the studies are complete, the study information along with other information on the drug is submitted to and reviewed by a government agency (e.g., the United States Food and Drug Administration, or FDA). If the data shows the study treatment is safe and effective, it may be approved for use by prescription.

This trial was ended abruptly in July 2016 when BioMarin decided they wouldn't earn enough money from the drug.

BioMarin News (BioMarin are the producers of BMN-701)

BMN-701:Potentially More Effective Treatment Option for Pompe - Dec 13 2010
This webinar talks about the BMN-701 drug treatment for Pompe Disease.  It takes around an hour to watch.  It is very interesting and gives a lot of hope for the future treatment of Pompe.
Photo used under Creative Commons from wotthe7734