Safety and efficacy of leriglitazone for preventing disease progression in men with adrenomyeloneuropathy (ADVANCE): a randomised, double-blind, multi-centre, placebo-controlled phase 2–3 trial

The brain penetrant PPARγ agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy

(2021) Laura Rodríguez-Pascau et al.   Science Translational Medicine

Gait Difficulties and Postural Instability in Adrenoleukodystrophy

(2021) Neha P. Godbole et al.   Frontiers in Neurology

Sensorimotor outcomes in adrenomyeloneuropathy show significant disease progression

(2021) Jennifer L. Keller et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Clinical and radiographic course of arrested cerebral adrenoleukodystrophy

(2020) Eric J. Mallack et al.   NEUROLOGY

Postural Body Sway as Surrogate Outcome for Myelopathy in Adrenoleukodystrophy

(2020) Wouter J. C. van Ballegoij et al.   Frontiers in Physiology

PPAR gamma agonist leriglitazone improves frataxin-loss impairments in cellular and animal models of Friedreich Ataxia

(2020) Laura Rodríguez-Pascau et al.   NEUROBIOLOGY OF DISEASE

Allogeneic hematopoietic stem cell transplantation with myeloablative conditioning for adult cerebral X-linked adrenoleukodystrophy

(2019) Nils Waldhüter et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The Landscape of Hematopoietic Stem Cell Transplant and Gene Therapy for X-Linked Adrenoleukodystrophy

(2019) Eric J. Mallack et al.   Current Treatment Options in Neurology

Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation

(2018) Gerald V. Raymond et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Progression of myelopathy in males with adrenoleukodystrophy: towards clinical trial readiness

(2018) Irene C Huffnagel et al.   BRAIN

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

(2015) Manuel Haas et al.   NEUROMUSCULAR DISORDERS

Frequent occurrence of cerebral demyelination in adrenomyeloneuropathy

(2014) M. de Beer et al.   NEUROLOGY

Severity score system for progressive myelopathy: development and validation of a new clinical scale

(2012) R.M. Castilhos et al.   BRAZILIAN JOURNAL OF MEDICAL AND BIOLOGICAL RESEARCH

X-linked adrenoleukodystrophy (X-ALD): clinical presentation and guidelines for diagnosis, follow-up and management

(2012) Marc Engelen et al.   Orphanet Journal of Rare Diseases

Evidence for a two-stage disability progression in multiple sclerosis

(2010) E. Leray et al.   BRAIN

Development and Validation of the Short Form of a Urinary Quality of Life Questionnaire: SF-Qualiveen

(2008) Véronique Bonniaud et al.   JOURNAL OF UROLOGY