US-based CHOP launches a leukodystrophy centre for children
US-based Children's Hospital of Philadelphia (CHOP) has launched a Leukodystrophy Centre of Excellence for children with degenerative inherited white matter diseases. (Source: Hospital Management)
Source: Hospital Management - May 19, 2015 Category: Hospital Management Source Type: news
Breakthrough in understanding Canavan disease
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. Canavan disease is a type of leukodystrophy that is an incurable and progressively fatal neurological condition. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - April 27, 2015 Category: Science Source Type: news
Meet Fireman Sam, swim in the sea and find a cure for my rare disease: Heartbreaking bucket list of terminally ill toddler who will gradually lose the ability to walk, talk, see or hear
Twenty-month-old Zach Parnaby from County Durham was diagnosed with the terminal disease Krabbe Leukodystrophy, which will gradually cause him to lose the ability to walk, talk, see or hear. (Source: the Mail online | Health)
Source: the Mail online | Health - March 5, 2015 Category: Consumer Health News Source Type: news
Metachromatic Leukodystrophy Therapeutics Clinical Trials Market...
This report provides elemental information and data relating to the...(PRWeb July 27, 2014)Read the full story at http://www.prweb.com/releases/2014-global-metachromatic/leukodystrophy-review/prweb12047502.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - July 27, 2014 Category: Pharmaceuticals Source Type: news
New advances in study of megalencephalic leukoencephalopathy
Several forms of leukodystrophies, genetic degenerative disorders that affect the myelin, are associated with vacuolization of myelin sheaths that enwrap axons of central neurons. Megalencephalic leukoencephalopathy (MLC), caused by mutations in MLC1 and GlialCAM, is a rare disease that entails this type of vacuoles. To date, there is not any treatment for patients. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - March 25, 2014 Category: Science Source Type: news
Tactics Can Cut Infection in Leukodystrophies
AUSTIN, Texas (MedPage Today) -- It may be possible to reduce care-related infections in children with leukodystrophies using standardized prevention efforts in the hospital setting, researchers reported here. (Source: MedPage Today Infectious Disease)
Source: MedPage Today Infectious Disease - November 4, 2013 Category: Infectious Diseases Source Type: news
HIV Virus Used As Vector To Get Gene Therapy Into Six Children
The HIV Virus can be used to treat two severe genetic diseases, according to two studies published in the journal Science. Researchers from the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan, Italy, have revealed that the HIV virus can be used to treat metachromatic leukodystrophy, a disease impairing the fatty covering that acts as an insulator around nerve fibers and Wiskott-Aldrich Syndrome, an immune system deficiency reducing the ability to form blood clots... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - July 16, 2013 Category: Consumer Health News Tags: HIV / AIDS Source Type: news
Gene therapy using HIV helps children with fatal diseases, study says
Gene therapy researchers say they used a safe version of HIV to prevent metachromatic leukodystrophy and halt Wiskott-Aldrich syndrome in children.Italian researchers have used a defanged version of HIV to replace faulty genes — and eliminate devastating symptoms — in children suffering two rare and fatal genetic diseases. (Source: Los Angeles Times - Science)
Source: Los Angeles Times - Science - July 11, 2013 Category: Science Source Type: news
Genetics Home Reference: Pol III-related leukodystrophy
(Source: NLM General Announcements)
Source: NLM General Announcements - June 18, 2013 Category: Databases & Libraries Source Type: news
New Approach To Improving Treatment For Multiple Sclerosis And Other Conditions
Working with lab mice models of multiple sclerosis (MS), UC Davis scientists have detected a novel molecular target for the design of drugs that could be safer and more effective than current FDA-approved medications against MS. The findings of the research study, published online in the journal EMBO Molecular Medicine could have therapeutic applications for MS as well as cerebral palsy and leukodystrophies, all disorders associated with loss of white matter, which is the brain tissue that carries information between nerve cells in the brain and the spinal cord... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - May 21, 2013 Category: Consumer Health News Tags: Multiple Sclerosis Source Type: news
How Turning Down Synthesis Of A Protein Improves Nerve, Muscle Function In Common Neuropathy
A potential new treatment strategy for patients with Charcot-Marie-Tooth disease is on the horizon, thanks to research by neuroscientists now at the University at Buffalo's Hunter James Kelly Research Institute and their colleagues in Italy and England. The institute is the research arm of the Hunter's Hope Foundation, established in 1997 by Jim Kelly, Buffalo Bills Hall of Fame quarterback, and his wife, Jill, after their infant son Hunter was diagnosed with Krabbe Leukodystrophy, an inherited fatal disorder of the nervous system. Hunter died in 2005 at the age of eight... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - April 30, 2013 Category: Consumer Health News Tags: Neurology / Neuroscience Source Type: news
Skin Cells Turned Directly Into The Cells That Insulate Neurons In Mouse Model
Researchers at the Stanford University School of Medicine have succeeded in transforming skin cells directly into oligodendrocyte precursor cells, the cells that wrap nerve cells in the insulating myelin sheaths that help nerve signals propagate. The current research was done in mice and rats. If the approach also works with human cells, it could eventually lead to cell therapies for diseases like inherited leukodystrophies - disorders of the brain's white matter - and multiple sclerosis, as well as spinal cord injuries. The study was published online in Nature Biotechnology... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - April 17, 2013 Category: Consumer Health News Tags: Stem Cell Research Source Type: news
Study Using Cells Forged From Human Skin Shows Promise In Treating MS, Myelin Disorders
A study out today in the journal Cell Stem Cell shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies. The study is the first successful attempt to employ human induced pluripotent stem cells (hiPSC) to produce a population of cells that are critical to neural signaling in the brain. In this instance, the researchers utilized cells crafted from human skin and transplanted them into animal models of myelin disease... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - February 11, 2013 Category: Consumer Health News Tags: Multiple Sclerosis Source Type: news
Cells forged from human skin show promise in treating multiple sclerosis, myelin disorders
Human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - February 7, 2013 Category: Science Source Type: news
Cells forged from human skin show promise in treating MS, myelin disorders
(University of Rochester Medical Center) A study out today in the journal Cell Stem Cell shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - February 7, 2013 Category: Global & Universal Source Type: news
