Global Study of Del-desiran for the Treatment of DM1

Conditions de participation

• Sexe:

  ALL

• Âges admissibles:

  16 to 65

Critères de participation

Key Inclusion Criteria:

* Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
* Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening

Key Exclusion Criteria:

* Breastfeeding, pregnancy, or intent to become pregnant during the study
* Unwilling or unable to comply with contraceptive requirements
* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
* Diabetes that is not adequately controlled
* History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
* Body Mass Index \> 35 kg/m2 at Screening
* Recently treated with an investigational drug or biological agent
* Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.

Note: Additional protocol defined Inclusion and Exclusion criteria apply