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Clinical Trials for Pompe Disease

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

Avrobio
Sanofi-Genzyme - neoGAA
​Duke
Amicus Therapeutics
Amicus Therapeutics
​Audentes Therapeutics
​Etubics
Spark Therapeutics
Lacerta
Oxyrane​
Pharming

​
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.


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  • Home
  • Our People
    • Our Patients >
      • Freda
      • Allyson
      • Christine
      • Dean
      • Samantha
      • Yakuta
      • Laurie
  • News and views
    • Press Releases
    • Personal Media Stories
    • Blog
    • Allyson's Florida Blog
    • Success Stories >
      • Maryze
      • Brad
      • Monique
      • Ben
      • Brian
  • About Pompe
    • What is Pompe Disease?
    • Symptoms
    • Diagnosis
    • Treatments
    • Recommended Links
  • Clinical Trials
  • Air Travel
  • Donations & Fund Raising
  • Glossary
  • Accessible NZ
  • NZPN Membership
  • NZPN Conference 2018 Summary
  • PHARMAC & Govt
    • Party stances on funding Rare Diseases
  • Contact us