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Studies for myasthenia gravis treatment

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Overview of clinical trials and how to join the studies

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Many new medications are being studied for the treatment of myasthenia gravis especially the disease modifying drugs. Disease modifying means that a medication treats the condition itself, not just the symptoms. Any new medication is approved only after it has been very carefully studied in clinical trials. 

Clinical trials are research studies that test how safe a drug is, how well it works, what the right dose is, and what the side effects are. They are overseen by the U.S. Food and Drug Administration (FDA). Every prescription medication you take passed the test and got approved by the FDA.

Clinical trials are designed to help doctors and scientists learn:

  • How well the study drug works compared to the standard treatment. Some patients get the study drug, some patients get the standard medication. You usually don’t know which one you get.
  • How well the study drug works compared to a placebo. A placebo looks like the study drug, but it is not a medication. Some patients get the study drug, some patients get the placebo. You usually don’t know which one you get.
  • If the study drug that is approved for one condition is also helpful for another condition.


You may benefit from being part of a clinical study by:

  • Getting a drug to treat your condition before it is ready for the public.
  • Giving you another chance to find a drug that may work for you if the available ones didn’t work or you couldn’t tolerate them.
  • Helping other patients who have or may get the same condition.


There are very strict rules that doctors and scientists have to follow when they are doing a clinical trial. If you join a study, they have to tell you exactly what the study is about, how it may help you, what the possible risks and side effects are, and what your responsibilities will be. A clinical trial is always voluntary, and you can change your mind any time.

If you are thinking about taking part in a clinical trial, learn as much as you can about the study before you decide to join. Here are some important questions to ask:

  1. What is the purpose of the study?
  2. What kinds of tests and treatments will I get? 
  3. What are the possible benefits for me to be in this study?
  4. What are the possible risks for me if I am in this study?
  5. How could the clinical trial affect my daily life?
  6. What side effects can I expect from the clinical trial? (Remember, there can be side effects from the standard treatments, too.)
  7. How long will the clinical trial last?
  8. How will my treatment change if I decide to drop out of the trial?


There are many studies going on with myasthenia gravis. Visit clinicaltrials.gov to learn about clinical trials near you. You can also ask your care team to learn more. 


Disclaimers:

This information is not a substitute for medical advice or treatment. Talk to your doctor or health care provider about your medical condition and prior to starting any new treatment. Coram assumes no liability whatsoever for the information provided or for any diagnosis or treatment made as a result. 

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