Adrenoleukodystrophy News

July 8, 2020July 8, 2020

Prenatal Genetic Diagnosis for Adrenoleukodystrophy

If you know that you and your partner are carriers of adrenoleukodystrophy (ALD) and risk passing the disease to your children, you may be considering a prenatal genetic diagnosis. What ... Read more

July 1, 2020July 2, 2020

What to Expect of a Physiotherapy Session for ALD Patients

Adrenoleukodystrophy News

Physiotherapy can be helpful to people with adrenoleukodystrophy (ALD), a rare genetic disease characterized by a buildup of toxic fatty molecules within cells. While there is currently no cure for ALD, ... Read more

June 29, 2020June 30, 2020

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

Adrenoleukodystrophy 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 (vorinostat) ... Read more

June 24, 2020June 24, 2020

Reproductive Options for Adrenoleukodystrophy Carriers

Adrenoleukodystrophy News

If you know you are a carrier of adrenoleukodystrophy (ALD), or if you or your partner has ALD, you may want to speak with a genetic counselor about your pregnancy ... Read more

June 15, 2020June 15, 2020

Autobahn Raises $76M to Advance ABX-002 Candidate for CNS Disorders

Adrenoleukodystrophy News

Autobahn Therapeutics has raised $76 million in Series B funding to advance the development of ABX-002, the company’s lead therapeutic candidate for adrenomyeloneuropathy (AMN) and multiple sclerosis (MS). ABX-002 is ... Read more

June 10, 2020June 10, 2020

Clinical Trials for Adrenoleukodystrophy

Adrenoleukodystrophy News

Developing treatments for rare diseases such as adrenoleukodystrophy (ALD) is extremely challenging. The small number of patients worldwide makes it difficult for researchers to develop and test new treatments. One way ... Read more

June 1, 2020June 2, 2020

ALD May Underlie Unexplained Adrenal Insufficiency in Boys, Case Report Shows

Adrenoleukodystrophy News

Boys with unexplained adrenal insufficiency should be screened for adrenoleukodystrophy (ALD), and repeated blood testing for very-long chain fatty acids (VLCFAs) may be key to its diagnosis, a case report suggests. ... Read more

June 1, 2020June 3, 2020

Adrenoleukodystrophy and Vision

Adrenoleukodystrophy News

Adrenoleukodystrophy (ALD) is a rare genetic disease that can lead to neurodegeneration (nerve cell death) and adrenal insufficiency, among other symptoms. However, symptoms vary widely between types of ALD. Cerebral ALD, ... Read more

May 26, 2020May 27, 2020

ALD Testing Should Be Done in Males with Primary Adrenal Insufficiency of Unknown Cause, Case Report Contends

Adrenoleukodystrophy News

Lack of testing for adrenoleukodystrophy (ALD) in males who have primary adrenal insufficiency due to an unknown cause may lead to a delayed or missing diagnosis of ALD. That finding ... Read more

May 26, 2020May 27, 2020

Seizures in Adrenoleukodystrophy and How to Deal with Them

Adrenoleukodystrophy News

Adrenoleukodystrophy (ALD) is a rare genetic disease characterized by the buildup of saturated very-long-chain fatty acids (VLCFAs) inside cells. When the disease affects the brain, as is most often does ... Read more

May 19, 2020May 20, 2020

First Patient Dosed in NEXUS Phase 2 Trial of Leriglitazone in CALD

Adrenoleukodystrophy News

A Phase 2 trial evaluating leriglitazone, Minoryx Therapeutics‘ investigational oral therapy for boys with cerebral adrenoleukodystrophy (CALD), has dosed its first patient at Hospital Sant Joan de Déu in Spain, the company announced. ... Read more

May 12, 2020May 13, 2020

New Agreement to Expand Production Capacity for Lenti-D for CALD, Other Gene Therapies

Adrenoleukodystrophy News, COVID-19 News

Hitachi Chemical Advanced Therapeutics Solutions and Apceth Biopharma GmbH, both subsidiaries of Hitachi Chemical Company, recently signed a new long-term development and manufacturing agreement with Bluebird Bio. The agreement will expand ... Read more

May 11, 2020May 20, 2020

Adrenoleukodystrophy: What Are the First Signs?

Adrenoleukodystrophy News

Some states in the U.S. test newborns for adrenoleukodystrophy (ALD). This often is the first indication to parents that their child may have ALD. However, there are different types of ... Read more

May 5, 2020May 5, 2020

SwanBio Raises $52M to Support Studies of Gene Therapy for Adult-onset ALD

Adrenoleukodystrophy News

SwanBio Therapeutics announced that it raised $52 million to help advance its viral-based gene therapy for adult-onset adrenoleukodystrophy (ALD) in studies essential for clinical testing, and to expand the company’s portfolio to ... Read more

April 28, 2020April 30, 2020

Case Report Describes Novel ALD-causing Gene Mutation

Adrenoleukodystrophy News

A new adrenoleukodystrophy-causing mutation in the ABCD1 gene was described in the case report of a 20-year-old Korean man. The report, “Novel ABCD1 Gene Mutation in a Korean Patient with X-Linked Adrenoleukodystrophy ... Read more