Posts by: Patricia Inacio, PhD

Patricia Inacio, PhD

Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.

October 14, 2020October 14, 2020

Eli-cel Gene Therapy Continues to Show Encouraging Results for Boys with CALD, Clinical Data Show

News

Data from the Starbeam Phase 2/3 trial continues to support the safety and effectiveness of the gene therapy candidate elivaldogene autotemcel (eli-cel, formerly known as Lenti-D) for the treatment of cerebral ... Read more

June 29, 2020June 29, 2020

Certain Epigenetic Medicines May Help Compensate for ABCD1 Gene Deficiency, Study Suggests

News

Treatment with certain epigenetic medicines may help compensate for a deficiency in the ABCD1 gene in patients with adrenoleukodystrophy (ALD), a study suggests. Researchers found Merck’s epigenetic modifier Zolinza ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

February 25, 2020February 25, 2020

New Worm Model May Help in More Quickly Finding Ways to Treat X-ALD, Study Shows

News

A new genetically modified earthworm model, Caenorhabditis elegans (C. elegans), mimics the features of human X-linked adrenoleukodystrophy (X-ALD), and could help in more quickly finding targets for therapies and at lower cost, a ... Read more

February 4, 2020February 4, 2020

Blood Levels of 3 Biomarkers May Help Identify Newborns at Risk for X-ALD, Chinese Study Suggests

News

Measuring the levels of three biomarkers in the blood may help identify newborns at risk of X-linked adrenoleukodystrophy (X-ALD), a Chinese study suggests. The study, “Evaluation of a panel ... Read more

October 22, 2019October 22, 2019

Immune Cells in Blood Samples from X-ALD Patients May Predict Response to Treatment, Study Says

News

Immune cells derived from blood samples of people with adrenoleukodystrophy (X-ALD) may help in evaluating their inflammatory status of patients and likely response to treatment, an early study suggests. The ... Read more

August 6, 2019August 6, 2019

First Clinical Trial of VK0214 to Treat X-linked ALD Planned for Early 2020, Viking Says

News

Viking Therapeutics plans to launch a first clinical trial for VK0214, its investigational oral small molecule to treat adrenoleukodystrophy in 2020, the company announced. Adrenoleukodystrophy (ALD), also known as X-linked ALD (X-ALD), is caused ... Read more

July 2, 2019July 2, 2019

Screening, Early Monitoring of Adrenal Glands Help Identify ALD in Newborns, Report Says

News

Newborn screening for adrenoleukodystrophy (ALD), including the monitoring and treatment of poor function of the adrenal glands, can prevent the development of potential life-threatening symptoms, a case series study ... Read more

June 11, 2019June 11, 2019

Combo of Antioxidants a Potential Therapy for Adrenomyeloneuropathy, Trial Suggests

News

Targeting oxidative stress with antioxidants holds promise to improve symptoms of adrenomyeloneuropathy (AMN), a pilot Phase 2 study suggests. The results show that treatment with a combo of antioxidants ... Read more

May 14, 2019May 14, 2019

#AANAM – High-dose Biotin Capsule Shows Promise in X-ALD

News

MD1003, a high-dose biotin, rescued locomotor (movement) activity and halted axon (nerve fiber) degeneration in two mouse models of X-linked adrenoleukodystrophy (ALD), including one with more severe and early ... Read more

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