Batten Disease News and Research

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Batten disease (also known as Spielmeyer-Vogt-Sjögren-Batten disease) is a rare, fatal autosomal recessive neurodegenerative disorder that begins in childhood. It is the most common form of a group of disorders called neuronal ceroid lipofuscinosis (or NCLs).

Raptor Pharmaceutical updates on cysteamine bitartrate Phase 3 clinical trial in cystinosis patients

Raptor Pharmaceutical Corp.,provides updates on timelines related to its clinical programs including the pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

24 Aug 2010

StemCells second-quarter net loss decreases to $4.61 million

StemCells, Inc., a leading stem cell company developing and commercializing novel stem cell therapeutics and tools for use in stem cell-based research and drug discovery, today reported financial results and business highlights for the second quarter ended June 30, 2010.

11 Aug 2010

NIH grants $14.85 million to complete two floors of NRI at Texas Children's Hospital

Neurological research at Baylor College of Medicine and Texas Children's Hospital is getting a big boost in the form of a $14.85 million grant from the National Institutes of Health to complete two floors of the Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital.

29 Jul 2010

BDSRA designates Nationwide Children's Hospital as Batten Disease Center of Excellence

Nationwide Children's Hospital has been selected as a Center of Excellence by the Batten Disease Support and Research Association (BDSRA), the largest research and support organization in North America for families that have children with Batten disease. BDSRA chose Nationwide Children's because of its clinical and research history with the disease and its ability to provide comprehensive care for these unique children.

23 Jul 2010

Raptor initiates enrollment in pivotal Phase 3 clinical trial of DR Cysteamine for nephropathic cystinosis

Raptor Pharmaceutical Corp. announced that it has enrolled the first patient in a pivotal Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

28 Jun 2010

Raptor Pharmaceutical acquires exclusive license to intellectual property for Huntington's Disease treatment

Raptor Pharmaceutical Corp., announced that the Company has acquired an exclusive worldwide license to intellectual property related to the potential treatment of Huntington's Disease from the Weizmann Institute of Science in Israel and Niigata University in Japan. In addition, Raptor has added Professor Lawrence Steinman, M.D., an inventor on the Weizmann patent, to its Advisory Board.

2 Jun 2010

STEM reports net loss of $6,124,000 for first-quarter 2010: MicroStockProfit.com

MicroStockProfit.com announces an investment report featuring StemCells Inc. The report includes financial, comparative and investment analyses, and industry information you need to know to make an educated investment decision.

5 May 2010

StemCells first-quarter total revenue up 307%

StemCells, Inc., a leading stem cell technology company with the dual focus of novel therapeutics and innovative research products, today reported financial results for the first quarter ended March 31, 2010 and recent business highlights.

5 May 2010

Neural stem cell transplantation promising for Batten disease

Neuronal Ceroid Liposfuscinosis (NCL), often referred to as Batten disease, is a rare and fatal neurodegenerative disorder that afflicts infants and young children. NCL is caused by a deficiency of a lysosomal enzyme, and is a relatively rare disease, affecting an estimated 2 to 4 of every 100,000 babies born in the U.S. Although NCLs are classified as rare diseases, they often affect more than one child in families that carry the defective gene, and tragically, are always fatal.

4 May 2010

StemCells to initiate second clinical trial of HuCNS-SC human neural stem cells in NCL

StemCells, Inc. announced today that it has submitted a protocol to the FDA for initiation of a second clinical trial of its proprietary HuCNS-SC human neural stem cells in neuronal ceroid lipofuscinosis, which is also often referred to as Batten disease.

22 Apr 2010

StemCells reports net loss of $5.23M for fourth-quarter fiscal 2009

StemCells, Inc. today reported financial results for the fourth quarter and year ended December 31, 2009. The Company's results include the operations of Stem Cell Sciences Plc (SCS) since April 1, 2009.

11 Mar 2010

Results of Phase 2a clinical trial of DR Cysteamine announced by Raptor Pharmaceutical

Raptor Pharmaceutical Corp., announced positive findings from the completed treatment phase of its open-label Phase 2a clinical trial of delayed-release cysteamine bitartrate ("DR Cysteamine") in adolescent patients with non-alcoholic steatohepatitis ("NASH"), a progressive form of liver disease believed to affect 2% to 5% of the U.S. population.

12 Oct 2009

Human neural stem cells to treat Pelizaeus-Merzbacher Disease

StemCells, Inc. (NASDAQ:STEM) announced today that it will soon initiate with the University of California, San Francisco (UCSF) Children’s Hospital a Phase I clinical trial to evaluate the therapeutic potential of StemCells’ proprietary HuCNS-SC® product candidate (purified human neural stem cells) to treat Pelizaeus-Merzbacher Disease (PMD), a myelination disorder that primarily affects infants and young children.

10 Sep 2009

StemCells’ novel neuroprotective approach for treating neurodegenerative diseases

StemCells, Inc. today announced the publication of preclinical data demonstrating for the first time that transplantation of its proprietary, purified human neural stem cells delays the loss of motor function in a mouse model of infantile neuronal ceroid lipofuscinosis (NCL).

4 Sep 2009

Febit's DNA-capture method selected by NCGR to re-sequence 400 genes involved in childhood diseases

The National Center for Genome Resources (NCGR) has selected febit's DNA-capture method, HybSelect, to re-sequence the exons of genes involved in over 400 of the most devastating childhood diseases with the ultimate goal of developing genetic tests to detect carrier status for recessive genetic diseases in prospective parents.

11 Aug 2009

Stemcells, Inc. reports Q2 financials

Results for the quarter reflect the Company’s acquisition of the operations of Stem Cell Sciences Plc (SCS), a UK-based company focused on cell-based technologies for drug discovery and screening.

10 Aug 2009

Launch of new foundation for Batten disease

Leading scientists, medical researchers and affected families joined together today to launch Beyond Batten Disease Foundation, an organization committed to finding a cure for this rare disease that claims the lives of children.

4 Mar 2009

New hope for Batten disease sufferers

Investigators at the University of Rochester Medical Center have uncovered a promising drug therapy that offers a ray of hope for children with Batten disease – a rare neurodegenerative disease that strikes seemingly healthy kids, progressively robs them of their abilities to see, reason and move, and ultimately kills them in their young twenties.

3 Jan 2008

New ray of hope for children with Batten disease

Investigators at the University of Rochester Medical Center have uncovered a promising drug therapy that offers a ray of hope for children with Batten disease

13 Dec 2007

Rochester receives $1.2 million to battle rare childhood disease

"For an orphan disease like Batten, which has such a clear-cut course of deterioration and no present cure, this grant is transformative both for these families and for us, as clinicians and researchers."

12 Sep 2007