Posts by: Jose Marques Lopes PhD

News

A new study in six Chinese men with adrenomyeloneuropathy (AMN) revealed a novel ABCD1 gene mutation, alterations such as skin hyperpigmentation, spasticity, and sexual dysfunction, as well as brain lesions in ... Read more

August 27, 2019August 27, 2019

Off-Label MS Therapy Rituximab Fails to Stop Progression of Advanced Childhood Cerebral ALD, Case Report Finds

News

Off-label use of the multiple sclerosis (MS) therapy rituximab (sold as Rituxan in the U.S. and MabThera in Europe) may not be recommended to treat patients with advanced stage childhood ... Read more

August 13, 2019August 13, 2019

Blood Stem Cell Transplant Feasible, but Better Before Onset of Neurological Complications, Case Report Shows

News

While allogeneic hematopoietic stem cell transplant (HSCT) is a feasible approach for people with adrenoleukodystrophy (ALD), it should be performed before the onset of neurological manifestations to effectively stop disease progression, ... Read more

June 25, 2019June 25, 2019

APOE4 Variant Linked to Poorer Neurological Outcomes in Boys with Cerebral ALD, Study Finds

News

Carrying the APOE4 gene variant is associated with greater disease severity, and poorer neurological function, in boys with cerebral adrenoleukodystrophy (cALD), a study found. The study, “Association between APOE4 and biomarkers in ... Read more

April 2, 2019April 2, 2019

Study Describes Two Atypical Cases of Adult-onset ALD

News

Two uncommon cases of adult-onset adrenoleukodystrophy (ALD) with congenital brain developmental defects and damage to small nerve fibers were described in a new study. The study, “Broadening the Spectrum of ... Read more

March 26, 2019March 26, 2019

20-Year Survival Seen in 73% of ALD and Other Metabolic Disorder Patients After Transplant, Study Reports

News

Twenty years after a blood or marrow transplant (BMT), patients with adrenoleukodystrophy (ALD) and other metabolic disorders can have a greater than 70% survival rate, according to a new study. ... Read more

January 25, 2019January 25, 2019

Leukodystrophy Diagnosis Less Likely in Children of Racial, Ethnic Minorities

News

Children and adolescents of racial or ethnic minorities, such as blacks and Hispanics, are less likely to be diagnosed with a leukodystrophy, according to a recent study. The research, “Association ... Read more