Status: Available
Expanded Access/Compassionate Use of Rugonersen in Patients With Angelman Syndrome
For a patient to be considered for this EAP, they must have consented to participate in the TANGELO Optional Open-label Extension (OOE), and continue to meet the inclusion/ exclusion criteria and have not received any other investigational drug for the treatment of AS between the last dose of the TANGELO OOE and the first dose in the EAP. The Physician making the request for expanded access considers continued treatment via the EAP with rugonersen to be a suitable treatment option for the patient under consideration.
Trial details
Biological sex: AllType: Expanded AccessSponsor: Oak Hill Bio LtdUpdated: Aug 22, 2025
Eligibility criteria
Signed informed consent must be obtained prior to the initiation of any study-re... [+19]