Effect of Oral Cimetidine in the Protoporphyrias

计划入组 26 人 · 首次发布于 2021 年 8 月 25 日

Oral Iron for Erythropoietic Protoporphyrias

计划入组 16 人 · 首次发布于 2016 年 12 月 1 日

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria

计划入组 184 人 · 首次发布于 2020 年 5 月 26 日

SMN1 gene modulator (X-linked infantile spinal muscular atrophy) (Recursion)

LV-ABCD1

RS1 gene therapy (AAV, X-linked retinoschisis)

Molecule X LLC

Mexico

SFJ Pharmaceuticals X, LTD.

X-Pert-Med GmbH

Germany

Disc Medicine Gains FDA Alignment on Bitopertin Trial Design for Erythropoietic Protoporphyria

Disc Medicine Advances Hematology Pipeline with Bitopertin NDA Plans and Phase 2 Trials

Disc Medicine Announces End-of-Phase 2 Meeting with FDA for Bitopertin in Erythropoietic Protoporphyria