4 results
To evaluate the efficacy of VX-121/TEZ/D-IVA in CF subjects who are homozygous forF508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, orhave at least 1 other TCR CFTR mutation and no F508del mutation
The primary objective of this study is to assess the number of detected pheochromocytoma lesions, proved by histology, with [18F]mFBG PET-CT compared to the conventional CT.
Primary ObjectiveTo evaluate the long-term safety and tolerability of VNZ/TEZ/D-IVA in subjects with CFSecondary ObjectiveTo evaluate the long-term efficacy of VNZ/TEZ/D-IVA
Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy for Seizures in Patients with Rare Seizure Disorders Such as Epileptic Encephalopathies Including Dravet Syndrome and Lennox-Gastaut Syndrome