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Lysosomal in Medical News

Scripps scientists find calcium channel blockers help normalize lysosomal storage disease cells

The study was published online February 5 in the February 2008 edition (Volume 6, Issue 2) of the journal PLoS Biology. The research team found that the hypertension drugs diltiazem and verapamil increased the overall function of mutant lysosomal enzymes associated with the conditions Gaucher d...

New DNA Tests Advances Treatment of Lysosomal Storage Diseases

A new set of laboratory tests using gene sequencing is able to help confirm 24 lysosomal storage diseases, providing physicians and patients the tools for much more accurate and rapid diagnosis. Lysosomal storage diseases are a group of more than 40 inherited and potentially life-threatening disor...

Patient Enrollment Complete for Phase I/II Clinical Trial for GALNS for Morquio A Syndrome

...-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syn...ient activity of N-acetylgalactosamine 6-sulfatase (GALNS)causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic s...

Tips from the American Journal of Pathology

...n cells. Treatment with gangliosides reversed the lysosomal disruption, which suggests that gangliosides protect against the lysosomal damage of a-synuclein accumulation. Based on th... M: Protective role of endogenous gangliosides for lysosomal pathology in a cellular model of synucleinopathies...

Cathepsin B increases apoptosis in fulminant hepatic failure

...ticle further indicated that LPS/D-Gal N-mediated cathepsin B expression initiates hepatocyte apoptosis in fulminant hepatic failure. Cathepsin B, a lysosomal cysteine protease, is a candidate for an apoptotic mediator originating from acidic vesicles. CA-074me is a selective inhibitor of cathepsin B , and i...

BioMarin Announces Initiation of Clinical Assessment Program for Morquio A Syndrome

...BioMarin Pharmaceutical Inc. (Nasdaq: BMRN ) announced today the initiation of the Morquio Clinical Assessment Program (MorCAP) for patients with the lysosomal storage disease Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. MorCAP is designed to augment available data on the disease by measur...

St. Jude Study Reveals a New Function for an Old Enzyme in Fatal Childhood Disease

...environment in a highly regulated process known as lysosomal exocytosis. These bags of enzymes, called lysosome... NEU1 regulates the pool of lysosomes destined for lysosomal exocytosis is by cutting off a sugar called sialic...omes to dock at the cell membrane and to engage in lysosomal exocytosis into the bone marrow environment. "Th...

Preliminary Results from Phase 2 Genzyme Study Highlight Potential of Novel Oral Compound for Gaucher Disease

... to develop an oral therapy for Gaucher disease given the remarkable impact that Cerezyme has had," said David P. Meeker, M.D., Genzyme's president of lysosomal Storage Disorder Therapeutics. "We are excited by the potential of Genz-112638. The data we collect from this study and from the Phase 3 program will ...

VIDEO from Medialink and Shire plc: Rare Diseases Must Be Top of the Health Agenda

...men with Fabry disease, compared with the general population. Fabry disease is one of a group of 45 similar genetic disorders, known collectively as lysosomal storage disorders. European Rare Disease Day is designed to improve the understanding, care and treatment of these kinds of diseases to improve the ...

Genzyme Delivers Strong Fourth Quarter to Conclude Outstanding Year

...t has been the most rapid for any of Genzyme's lysosomal storage disorder treatments. In December, Gen...the first quarter of 2009. Newborn Screening for lysosomal storage disorders -- Based on its belief that ea...an lead to improved outcomes for patients with lysosomal storage disorders, and as part of its commitme...
Lysosomal in Medical Technology

Amicus Therapeutics Commences Phase 3 Trial Evaluating Amigal(TM) for the Treatment of Fabry Disease

... About Fabry Disease Fabry disease is a lysosomal storage disorder caused by inherited genetic mutat...e A (a-Gal A). Deficient a-Gal A activity leads to lysosomal accumulation of globotriaosylceramide (GL-3), whic...three lead pharmacological chaperone compounds for lysosomal storage disorders, Amigal, Plicera and AT2220. Und...

BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome

...-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syn...ent activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic s...

Amicus Therapeutics Announces Issuance of New Plicera(TM) Composition of Matter Patent

...nd commercialize Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the collaboration, Shire received commercial right...ding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet me...

Amicus Therapeutics Suspends Enrollment for Phase 2 Clinical Trial of AT2220 for Pompe Disease

...nd commercialize Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire received commercial rights ou...ding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet me...

Amicus Therapeutics Announces Positive Progress of Three Lead Clinical Programs and General Outlook for 2009

...e progress in its three lead clinical programs for lysosomal storage disorders and presented a corporate outloo... increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genet...three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire rece...

Raptor Pharmaceuticals to Collaborate with Centre Hospitalier Universitaire d'Angers for Phase II Clinical Trial in Huntington's Disease

...e Cysteamine is approved for sale by the FDA and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), a rare, genetic lysosomal storage disease. DR Cysteamine is designed as an enterically coated, proprietary oral formulation of cysteamine to potentially reduce dosing frequency...

Raptor Pharmaceuticals Announces Phase 2a Clinical Trial Initiation in Non-Alcoholic Steatohepatitis (NASH)

...affected patients. About Cysteamine and DR Cysteamine Cysteamine is cleared for market by the FDA and EMEA to treat cystinosis, a rare and genetic lysosomal storage disease. The drug's potential to accumulate in the liver may create a targeted treatment effect for patients with NASH. DR Cysteamine is desig...

Amicus Therapeutics Announces Second Quarter 2008 Financial Results

...t a broader range of human genetic diseases beyond lysosomal storage diseases. As part of this effort, Amicus c...three lead pharmacological chaperone compounds for lysosomal storage disorders, Amigal, Plicera and AT2220. In ... increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genet...

Amicus Therapeutics Announces Successful Completion of End of Phase 2 Meeting With FDA for Amigal in Fabry Disease

...three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire rece...oral therapy for the treatment of Fabry disease, a lysosomal storage disorder, which can cause damage to specif... increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genet...

Raptor Pharmaceuticals Enters Collaboration Agreement with UC San Diego in Liver Disease

...ation ("FDA") for the treatment of nephropathic cystinosis, a rare, genetic lysosomal storage disease. Raptor is currently developing DR Cysteamine, a proprietar...European Medicines Agency to treat nephropathic cystinosis, a rare, genetic lysosomal storage disease. DR Cysteamine is designed as an improved, enterically coat...
Lysosomal in Medical Products

Leukocyte Lysosomal Enzyme

Description: ARUP Laboratories is a national reference laboratory and a worldwide leader in innovative laboratory research and development. ARUP offers an extensive test menu of highly complex and unique medical tests in clinical and anatomic pathology. Owned by the University of Utah, ARUP Laboratories' client...
Company:ARUP Laboratories
Lysosomal in Medical Dictionary

Tay-sachs

...ultimate elimination of Tay - Sachs disease, Canavan disease, and related lysosomal storage diseases. National Tay - Sachs and Allied Diseases Associatio...and programs for families with children affected by Tay - Sachs and other lysosomal storage disorders. Tay - Sachs disease is a progressive neurologica...

Tay-Sachs disease

...dants of Central and Eastern ... Dedicated to the prevention and ultimate elimination of Tay - Sachs disease , Canavan disease , and related lysosomal storage diseases. In late-onset Tay - Sachs disease (LOTS), the hex A gene is altered so that it ... A baby with Tay - Sachs disease app...

Scheie syndrome

...; Seek Foundation, Finding Cures for Children with lysosomal Disease. ... The clinical features of Scheie Sy... Scheie syndrome are missing a substance called lysosomal alpha-L-iduronidase. ... ... Pediatric Databas... Scheie syndrome are missing a substance called lysosomal alpha ... Learn about Scheie syndrome ca...

Sanfilippo syndrome

... Sanfilippo syndrome , or Mucopolysaccharidosis III (MPS-III) is a rare autosomal recessive lysosomal storage disease caused by a deficiency in one of the enzymes needed to break down the glycosaminoglycan heparan sulfate . Sanfilippo syndro...

Lipofuscin

... Lipofuscin is the name given to finely granular yellow-brown pigment granules composed of lipid-containing residues of lysosomal digestion. It is considered one of the aging or "wear and tear" pigments, found in the liver, kidney, heart muscle, adrenals, nerve ce...

Hurler syndrome

...urler syndrome do not make a substance called lysosomal alpha-L-iduronidase. ... Hurler's syndrome ...urler syndrome do not make a substance called lysosomal alpha ... Information about Hurler syndro...urler syndrome do not make a substance called lysosomal alpha-L-iduronidase. ... Free articles and mul...

Hunter syndrome

... Hunter syndrome , or mucopolysaccharidosis Type II, is a lysosomal storage disease caused by a deficient (or absent) enzyme, iduronate-2-sulfatase (I2S). :544 The syndrome is named after physician Charles A. Hu...

Exudate

... ... Wound Care Information Network. Allevyn Gentle Advertisement - Available Now. Exudate ... Exudate contains various components, including: lysosomal enzymes, WBC's, ... Online Medical Dictionary and glossary with medical definitions ... Exudate : A fluid rich in protein and cellular elements...

Cystinosis

... Cystinosis is a lysosomal storage disease characterized by the abnormal accumulation of the amino acid cystine. It is a genetic disorder that typically follows an autoso...
Lysosomal in Biological News

Proteomics study yields clues as to how tuberculosis might be thwarting the immune system

...ith digestive enzymes from another cell organelle, called a lysosome, which fuses with the phagosome. Macrophages, like other kinds of cells, also use lysosomal enzymes for internal housekeeping. However, until now there has been no direct biochemical evidence of a link between phagocytosis and autophagy. W...

In scientific first, Einstein researchers correct decline in organ function associated with old age

...ng in a buildup of undigested proteins within the cells. She also detected the primary cause for this age-related decline: a fall-off in the number of lysosomal receptors capable of binding chaperones and their damaged proteins. Could replenishing lost receptors in older animals maintain the efficiency of this...

Gene therapy slows progression of fatal neurodegenerative disease in children

... (LINCL) is an autosomal recessive genetic disorder that causes degeneration of the central nervous system. It is a form of Batten disease, a group of lysosomal storage disease in which a lipofuscin-like material is not broken down and accumulates in neurons, causing cognitive impairment, visual failure, seizu...

Penn researchers link cell's protein recycling systems

...egeneration. They use the fly eye, a neuron-rich tissue, as a surrogate for the brain because it is easy to visualize. They discovered that making the lysosomal system more or less active dramatically influenced the severity of neurodegeneration. “We found that whenever we knocked the lysosome system dow...

Stem cells act through multiple mechanisms to benefit mice with neurodegenerative disease

...netic diseases that afflicts 1 in 5000 patients, typically children (called lysosomal storage diseases, described in more detail below), but which is often used ... one person in 5,000 is affected by a disease that falls into a category of lysosomal storage diseases. Currently there is no treatment for Sandhoff or ...

Dangerous tricksters: Some bacterie use immune cells to reproduce

...lopment inside the macrophage, preventing acidification and merging with the lysosomes. As a result the bacteria are not exposed to the large array of lysosomal digestive enzymes and acid. Killing the killer 'Basically what this means is that the rhodococci manipulate their host cell, they make it them...

Infants with Rare Genetic Disease Saved By Cord Blood Stem Cells

...in all. The oldest Duke patient transplanted for a lysosomal storage disease is now seven years old and has dev...niversal screening of all newborns for the genetic lysosomal storage diseases. The major barrier to universal n...esting itself would be relatively low. Although lysosomal storage diseases are rare ?fewer than 5,000 births...

Genetic therapy reverses nervous system damage in animal model of inherited human disease

...t one day treat an entire class of diseases called lysosomal storage disorders which cause severe, sometimes fa...the disease itself is rare, AMD is one of about 50 lysosomal storage diseases, a class of diseases that account...hs disease, Hunter disease and Pompe disease. In a lysosomal storage disease, cellular debris accumulates withi...

Infants With Rare Genetic Disease Saved by Cord Blood Stem Cells

...in all. The oldest Duke patient transplanted for a lysosomal storage disease is now seven years old and has dev...niversal screening of all newborns for the genetic lysosomal storage diseases. The major barrier to universal n...esting itself would be relatively low. Although lysosomal storage diseases are rare ?fewer than 5,000 births...

Current human embryonic stem cell lines contaminated UCSD/Salk team finds

...ey determined that Neu5Gc gets into human cells by being engulfed in fluid droplets and then moved to the cytoplasm of the cell by a "pump" called the lysosomal sialic acid transporter. Varki noted that this pathway is an unusual and previously unknown one that may also be relevant to the entry of other small ...
Lysosomal in Biological Technology

Amicus Therapeutics Announces Second Quarter 2009 Financial Results

...three lead pharmacological chaperone compounds for lysosomal storage disorders: Amigal, Plicera and AT2220. Und... increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genet...three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire rece...

Amicus Therapeutics Announces Second Quarter 2009 Financial Results Release Date

...ly bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. FOLD -G ...

Raptor Pharmaceuticals Doses First Patient in Phase 2b Clinical Trial of DR Cysteamine for Cystinosis

...aluate Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") capsules in nephropathic cystinosis ("cystinosis"), a rare genetic lysosomal storage disease. Under Raptor's open Investigational New Drug application ("IND"), UCSD is performing the Raptor-sponsored trial at its General Clinic...

Amicus Therapeutics to Present at the Bank of America and Merrill Lynch 2009 Health Care Conference on May 13, 2009

...ly bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. ...

Amicus Therapeutics Announces First Quarter 2009 Financial Results Release Date

...ly bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. FOLD -G ...

Amicus Therapeutics to Present at the 16th Annual Future Leaders in the Biotech Industry Conference

...y bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. FOLD -G ...

Amicus Therapeutics Presents Positive Results From Phase 2 Extension Study of Amigal(TM) for Fabry Disease at ACMG 2009 Annual Meeting

...three lead pharmacological chaperone compounds for lysosomal storage disorders. Under the agreement, Shire rece... About Fabry Disease Fabry disease is a lysosomal storage disorder caused by inherited genetic mutat...e A (a-Gal A). Deficient a-Gal A activity leads to lysosomal accumulation of globotriaosylceramide (GL-3), whic...

Publication of Shire plc's Annual Report 2008

... treatment of Metachromatic Leukodystrophy ('MLD') Shire has an ongoing enzyme replacement therapy program for the treatment of MLD, which is a lysosomal storage disorder that results from a deficiency in the enzyme arylsulfatase-A ('ASA'). In June 2008 Shire completed its acquisition from Zymenex of th...

BioMarin's Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA

...-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syn...ent activity of N-acetylgalactosamine 6-sulfatase (GALNS) causing excessive lysosomal storage of keratan sulfate (KS). This excessive storage causes a systemic s...

Amicus Therapeutics to Present at the Cowen and Company 29th Annual Healthcare Conference on March 17, 2009

...y bind to and stabilize proteins in cells, leading to improved protein folding and trafficking, and increased activity. Amicus is initially targeting lysosomal storage disorders, which are severe, chronic genetic diseases with unmet medical needs. FOLD -G ...
Lysosomal in Biological Products

Human Cathepsin D from Calbiochem

Description:... Lyophilized. Lyophilized from 2 mM sodium phosphate buffer, pH 6.5. Cathepsin D from human liver. Cathepsin D is a major lysosomal aspartyl protease in mammalian cells produced as a 52 kDa proenzyme. Overexpression of cathepsin D in human breast cancers is associated with higher r...
Company:Calbiochem
Lysosomal in Biological Definition

Disulfide bond

...re formed in the lumen of the RER (rough endoplasmic reticulum) but not in the cytosol . Thus disulfide bonds are found only in secretory proteins, lysosomal proteins, and the exoplasmic domains of membrane proteins. Disulphide bonds are also formed within and between protamines in the sperm chromatin...

Lipase

...iving cells to degrade lipids. In the example of lysosomal lipase, the enzyme is confined within an organell...ody , including pancreatic lipase, hepatic lipase, lysosomal lipase, hepatic lipase, gastric lipase, endothelia... are both caused by mutations in the gene encoding lysosomal lipase, also referred to as...
Lysosomal in Biological Dictionary

Lysosome

...ontains many enzymes (collectively ... The membrane of the lysosome has intergral proteins called hydrogen ion ATPases (proton pumps) ... ... lysosomal hydrolases are known and lysosomes have a great variety of morphologies and functions. ... GO:0005764 : lysosome [305 gene products] ... Ea...

Deoxyribonuclease

...ts. Histones. Lysozyme ... Cow protein-coding gene DNASE2. Represented by 147 ESTs from 44 cDNA libraries. ... Bos taurus deoxyribonuclease II, lysosomal (DNASE2), mRNA. PA ... Pig protein-coding gene DNASE2. Represented by 129 ESTs from 49 cDNA libraries. ... Sus scrofa deoxyribonuclease II mRNA...
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