Share your thoughts on gene therapy with the National Fragile X Foundation

“Gene therapy” seems to be all the buzz these days!  As gene therapies continue to enter rare disease and Fragile X spaces, we know that our community has become increasingly curious about what this means for Fragile X.

What are your thoughts and feelings on gene therapy?  We want to hear from YOU!

Gene therapy is a technique that targets a person’s genes to treat or cure a disease.  Gene therapies can work in different ways, such as:

  • Replacing a disease-causing gene with a healthy copy of the gene
  • Inactivating or “turning off” a disease-causing gene that is not working properly
  • Introducing a new or altered gene into the body to help treat a disease

The National Fragile X Foundation wants to know your thoughts and feelings on gene therapy.  We feel strongly that we have a duty to educate on the opportunities and realities of gene therapies, so our community can make informed decisions when the time comes to consider potential trials and treatments. 

While there are treatments available for Fragile X syndrome (FXS) now, they primarily work on targeting symptoms caused by FXS, and not the actual FXS itself (the genetic factors like CGG repeat number).  Since FXS is a genetic disorder, there’s interest to see if gene therapy may provide a direct approach to targeting the fundamental and genetic causes of FXS.

If you consider yourself to be part of the Fragile X community, we need you to complete this survey and share more about your thoughts and feelings regarding gene therapy.

This survey will take ~5 minutes to complete, and your responses will remain anonymous.  We will use the results of this survey to bring the most relevant, sought-after gene therapy content to the community.

Share your opinions by completing the survey.

Anna De Sonia, Director, Research Facilitation.

Anna De Sonia
Anna joined the NFXF team in 2024 as Director of Research Facilitation.  She has many years of research experience, starting as a clinical research coordinator at Rush University Medical Center in Chicago in 2010.  There she worked on a variety of clinical trials in the pediatric neurology division, specializing in Fragile X research. Anna earned her bachelor’s in psychology and is a certified clinical research coordinator (CCRC®) through the ACRP (Association of Clinical Research Professionals). She loves spending time with her dog, traveling and exploring new cultures, listening to music, and enjoying time with friends and family.