Canavan Disease News and Research

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Rowan University, Bamboo Therapeutics partner to combat Canavan disease

Rowan University and Bamboo Therapeutics, Inc. have entered into an asset transfer agreement to commercialize a novel gene therapy for the treatment of Canavan disease. A rare but devastating neurological disease that tragically takes a child's life by age 10, Canavan disease is one of the most common and complex degenerative cerebral diseases in infants.

17 Jul 2015

UC Davis researchers settle long-standing controversy surrounding Canavan disease

UC Davis investigators have settled a long-standing controversy surrounding the molecular basis of an inherited disorder that historically affected Ashkenazi Jews from Eastern Europe but now also arises in other populations of Semitic descent, particularly families from Saudi Arabia.

28 Apr 2015

RowanSOM researcher awarded NINDS grant to develop stem cell-based therapy for Canavan disease

Paola Leone, PhD, the director of the Cell and Gene Therapy Center and a professor of Cell Biology at the Rowan University School of Osteopathic Medicine, has been awarded a three-year, $477,000 grant from the National Institute of Neurological Disorders and Stroke to develop a stem cell-based therapy for Canavan disease, a rare but devastating neurological disorder in children that typically takes a child's life by age 10.

26 Feb 2015

Narrow therapeutic window for Canavan disease

Gene therapy for Canavan disease slows brain atrophy and stabilizes patients’ clinical condition, show findings that highlight the need for very early diagnosis and intervention.

22 Dec 2012

Novel virus-based gene therapy holds promise for Canavan disease

Research led by Paola Leone, PhD, of the University of Medicine and Dentistry of New Jersey-School of Osteopathic Medicine (UMDNJ-SOM), demonstrates the long term safety and benefit of a virus-based gene therapy that has been applied for the first time in a clinical setting.

21 Dec 2012

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder.

20 Dec 2012

FDA lifts hold, allows Geron to proceed with Phase I clinical trial of GRNOPC1 in spinal cord injury

Geron Corporation announced today that the U.S. Food and Drug Administration (FDA) has notified the company that the clinical hold placed on Geron's Investigational New Drug (IND) application has been lifted and the company's Phase I clinical trial of GRNOPC1 in patients with acute spinal cord injury may proceed.

30 Jul 2010

Gene patenting policies affect patient access to testing for disease-causing genes: Study

Policies allowing genes to be patented can make it more difficult for patients to access testing for important disease-causing gene mutations, according to a series of papers in a special online supplement published by Genetics in Medicine, the official peer-reviewed journal of The American College of Medical Genetics.

7 May 2010

Canavan Disease News and Research

Rowan University, Bamboo Therapeutics partner to combat Canavan disease

UC Davis researchers settle long-standing controversy surrounding Canavan disease

RowanSOM researcher awarded NINDS grant to develop stem cell-based therapy for Canavan disease

Narrow therapeutic window for Canavan disease

Novel virus-based gene therapy holds promise for Canavan disease

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

FDA lifts hold, allows Geron to proceed with Phase I clinical trial of GRNOPC1 in spinal cord injury

Gene patenting policies affect patient access to testing for disease-causing genes: Study

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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