Adrenoleukodystrophy News and Research

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Adrenoleukodystrophy (ALD) is one of a group of genetic disorders called the leukodystrophies that cause damage to the myelin sheath, an insulating membrane that surrounds nerve cells in the brain.

New C. elegans model will accelerate study of a rare disease

The model will permit to accelerate and reduce the price of the research, which is studying the mechanisms and the possible pharmacological targets for the neuronal alterations of this disease.

20 Feb 2020

Researchers make progress toward gene-based strategies to treat inherited neurometabolic diseases

Researchers are making great strides toward developing gene-based strategies to treat a variety of inherited neurometabolic diseases characterized by severe neurological involvement.

29 Oct 2019

Scientific breakthrough provides new hope for people living with multiple sclerosis

A scientific breakthrough provides new hope for millions of people living with multiple sclerosis. Researchers at Oregon Health & Science University have developed a compound that stimulates repair of the protective sheath that covers nerve cells in the brain and spinal cord.

19 Apr 2019

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Bluebird bio, Inc. and Gritstone Oncology, Inc. today announced a collaboration to research, develop and commercialize products for the treatment of cancer using cell therapy.

23 Aug 2018

Coconut oil diet increases vitality, lifespan of fruit flies with peroxisomal disorder

"Lorenzo's Oil" was to help a seriously ill boy suffering from a peroxisomal disorder (adrenoleukodystrophy/ALD). The true story was turned into a film which made the rare disease well known. Scientists from the University of Bonn, the German Center for Neurodegenerative Diseases and the German Cancer Research Center investigated such peroxisomal diseases on fruit flies.

20 Jun 2018

Moms of children with rare genetic illness push for wider newborn screening

Kerri De Nies received the news this spring from her son's pediatrician: Her chubby-cheeked toddler had a rare brain disorder.

5 Oct 2017

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

In a recent clinical trial, a gene therapy to treat cerebral adrenoleukodystrophy (CALD) -- a neurodegenerative disease that typically claims young boys' lives within 10 years of diagnosis -- effectively stabilized the disease's progression in 88 percent of patients, researchers from the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Massachusetts General Hospital report today.

4 Oct 2017

Viking submits IND application to FDA to conduct VK2809 Phase 2 study in patients with LDL-C and fatty liver disease

Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, today announced that it has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration to conduct a Phase 2 study of VK2809 in patients with hypercholesterolemia and fatty liver disease.

17 Nov 2015

$Viking begins VK5211 Phase 2 trial in patients recovering from hip fracture$

Viking begins VK5211 Phase 2 trial in patients recovering from hip fracture

Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, today announced the initiation of dosing in the company's Phase 2 clinical trial of VK5211 in patients who recently suffered a hip fracture.

3 Nov 2015

Viking successfully completes safety, tolerability and pharmacokinetic study of VK5211 in elderly subjects

Viking Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class or best-in-class therapies for metabolic and endocrine disorders, today announced the successful completion of a short-term safety, tolerability, and pharmacokinetic study of VK5211 in healthy elderly subjects. VK5211, the company's lead program for muscle and bone disorders, is an orally available, non-steroidal selective androgen receptor modulator (SARM) being developed for the treatment of patients recovering from non-elective hip fracture surgery.

20 Oct 2015

Drug used to control Type II diabetes can help repair spinal cords of mice suffering from inherited disease

A drug used to control Type II diabetes can help repair the spinal cords of mice suffering from the inherited disease adrenoleukodystrophy which, untreated, leads eventually to a paralysis, a vegetative state and death.

10 Jun 2013

IDIBELL signs patent licensing agreement with Minoryx

The Bellvittge Biomedical Research Institute has signed a licensing agreement with the Spanish biotechnology company Minoryx of a patent for the treatment of X-linked adrenoleukodystrophy, a rare serious neurodegenerative disease which has no effective treatment.

8 May 2013

bluebird bio completes $60 million Series D financing

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor.

25 Jul 2012

FDA, EMA grant orphan drug designation to bluebird bio's gene therapy product for adrenoleukodystrophy

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD).

19 Jun 2012

genOway acquires worldwide exclusive license for RMCE technology from bluebird bio for genetically modified rodents

genOway and bluebird bio report today genOway acquisition of an exclusive worldwide license for the RMCE technology (developed at the Massachusetts Institute of Technology) in the field of genetically modified rodents.

12 Jul 2011

bluebird bio announces receipt of additional $30 million in financing

bluebird bio, an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced that the company has secured an additional $30 million in financing.

20 Apr 2011

Children's Hospital pediatric immunologist collaborates with European gene therapy researchers to study WAS syndrome

A pediatric immunologist at The Children's Hospital of Philadelphia collaborated with European gene therapy researchers who achieved marked clinical improvements in two young children with Wiskott-Aldrich syndrome, a very rare but often severe immunodeficiency disorder.

16 Nov 2010

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.

16 Sep 2010

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis."

19 May 2010

Genetix Pharmaceuticals announces completion of $35M Series B financing

Genetix Pharmaceuticals, a leading gene therapy company developing breakthrough treatments for severe genetic disorders, announced today that it has completed a $35 million Series B financing with new investors Third Rock Ventures and Genzyme Ventures joining TVM Capital, Forbion and Easton Capital. Proceeds from the financing will be used to advance current clinical programs, strengthen platform capabilities and further expand the team.

12 Mar 2010