Posts by: Teresa Carvalho

News

Clinical trials testing Minoryx Therapeutics‘ leriglitazone (MIN-102), an investigational oral therapy for all forms of X-linked adrenoleukodystrophy (ALD) — including childhood cerebral ALD (CALD) and adrenomyeloneuropathy (AMN) — will receive ... Read more

October 7, 2020October 7, 2020

Approval of CALD Gene Therapy, Lenti-D, Under Review in Europe

News

The European Medicines Agency (EMA) accepted an application requesting approval of the gene therapy Lenti-D (elivaldogene autotemcel, or eli-cel) to treat cerebral adrenoleukodystrophy (CALD), its developer, Bluebird Bio, announced. If approved, the submission — in the ... Read more

September 23, 2020September 23, 2020

Phase 1 Study of VK0214 as Potential Oral Treatment for ALD to Open

News

Viking Therapeutics is launching a Phase 1 clinical trial testing increasing doses of VK0214, its oral investigative molecule to treat adrenoleukodystrophy (ALD), against a placebo in healthy people. “We are excited to ... Read more