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Tay-Sachs, Canavan, GM1 and Sandhoff diseases

Evaluating Experimental Therapies - US and Abroad

Details

GOALS

  • Families have the tools and resources to evaluate experimental therapies
  • Families feel supported in all care choices
  • Families feel empowered to make informed choices they can feel good about

RESOURCES

ClinicalTrials.gov
ClinicalTrials.gov offers up-to-date information for locating federally and privately supported clinical trials. Only clinical trials that meet the site's rigorous scientific standards are listed.

PubMed
PubMed provides free online searchs of peer reviewed original scientific papers.  Publishing work is an important part of the scientific process. It allows scientific peers to review the work and critique the approach. It is a concern when data is not published. Please contact the office at (617) 277-4463 or This email address is being protected from spambots. You need JavaScript enabled to view it. if you are interested in any particular paper. We are often able to access the full paper, and members of our Scientific Advisory Committee can provide insights into the quality of the paper, what the paper means in lay language and address the reputation of the publishing journal.

Ask the Experts
NTSAD is able to connect you directly with leading experts to provide their perspective. These world-class scientists will give you their expert, unbiased opinion on an experimental therapy so you are able to make the decision that is right for you and your family.

QuackWatch
QuackWatch is an international network of people concerned about health-related frauds, myths, fads, fallacies, and misconduct. Search the website for information on the experimental therapy you are considering.  

QUESTIONS TO ASK

Where is therapy offered?
A US doctor providing therapy outside the US bypasses the FDA's standards of safety and efficacy which are in place to protect patients. If the team offering the experimental therapy is so confident in their approach why bypass the FDA? Also ask who pays if there is a medical emergency while out of the country.

Is it IRB approved?
An Internal Review Board is an independent team of scientists and lay people who evaluate whether the potential benefits outweigh the potential risks.

How much does the experimental therapy cost?
Typically, there is little to no cost to participate in clinical trials that are IRB approved, and listed on ClinicalTrials.gov .

POTENTIAL RED FLAGS

  • Claims based on patient testimonials - Not reliable because sometimes patients want so much to believe that a treatment is helping them that they convince themselves that it has.

  • Multiple diseases treated with same treatment

  • Claims that there is no risk- There is always risk. Information about the possible risks should be available from pre-clinical research.

  • High cost of treatment or hidden costs - It is not customary for someone to pay to be in a clinical trial (other than travel and personal expenses). Consider whether you should pay for a treatment that is unproven. Also, ask about the costs of emergency medical care if something goes wrong, particularly if you are outside the US.

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