Question 1

What are the potential benefits and risks of participating in this trial?

Accepted Answer

This is a question you may want to discuss with your doctor regarding the A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 clinical trial (NCT06844214). Contact the study team or your healthcare provider for specific answers.

Question 2

How does SAR446268 compare to my current treatment options?

Accepted Answer

This is a question you may want to discuss with your doctor regarding the A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 clinical trial (NCT06844214). Contact the study team or your healthcare provider for specific answers.

Question 3

What kind of monitoring will I receive during the trial?

Accepted Answer

This is a question you may want to discuss with your doctor regarding the A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 clinical trial (NCT06844214). Contact the study team or your healthcare provider for specific answers.

Question 4

What are the possible side effects of this gene therapy?

Accepted Answer

This is a question you may want to discuss with your doctor regarding the A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 clinical trial (NCT06844214). Contact the study team or your healthcare provider for specific answers.

Question 5

How often will I need to visit the clinic during the study?

Accepted Answer

This is a question you may want to discuss with your doctor regarding the A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 clinical trial (NCT06844214). Contact the study team or your healthcare provider for specific answers.

Question 6

What happens if I experience side effects or complications during the trial?

Accepted Answer

This is a question you may want to discuss with your doctor regarding the A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 clinical trial (NCT06844214). Contact the study team or your healthcare provider for specific answers.

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

What this study is about

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

What this study is about