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Gaucher in Medical News

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

CAMBRIDGE, Mass., May 7 /PRNewswire-FirstCall/ -- Genzyme Corp. (Nasdaq: GENZ ) reported today that a preliminary analysis of data from an ongoing open-label Phase 2 clinical trial of its investigational oral therapy Genz-112638 showed that the compound produced a meaningful impact on key cli...

Genetic disease treatments and pharmacogenetics: From scientific discovery to medical delivery

...nzyme replacement therapy for children with Type I gaucher Disease, and new research findings that offer impo...ME REPLACEMENT THERAPY IN 884 CHILDREN WITH TYPE I gaucher DISEASE gaucher disease is a rare genetic disorder that occurs whe...

Genzyme Delivers Strong Fourth Quarter to Conclude Outstanding Year

... collaboration with Bayer Schering Pharma AG, Germany. Genz-112638 for gaucher disease -- Genzyme is investing in the development of an innovative, next- generation product for the treatment of gaucher disease. The company has completed enrollment in a phase 2 trial of the...

Protalix BioTherapeutics Appoints Professor Roger D. Kornberg to Board of Directors

...hase III clinical trial in Israel, the United States and other locations for its lead product candidate, prGCD, for its enzyme replacement therapy for gaucher disease, a lysosomal storage disorder in humans, and has reached an agreement with the United States Food and Drug Administration on the final design ...

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

...t lysosomal enzymes associated with the conditions gaucher disease, -mannosidosis, and type IIIA mucopolysacc...orage diseases. The most prevalent of these is gaucher disease, which is the most common genetic disorder...people of Eastern European ancestry. Patients with gaucher disease may bruise easily due to low blood platele...

Top Scientists to Receive Prestigious Awards From the American Society of Hematology

...an insight that has become one of the cornerstones of mammalian genetics. He also developed screening tests for the genetic disorders galactosemia and gaucher disease. He is currently Professor and Chairman, Department of Molecular and Experimental Medicine, The Scripps Research Institute, La Jolla, CA. ...

Protalix BioTherapeutics Announces Resignation of Board Members

...hase III clinical trial in Israel, the United States and other locations for its lead product candidate, prGCD, for its enzyme replacement therapy for gaucher disease, a lysosomal storage disorder in humans, and has reached an agreement with the United States Food and Drug Administration on the final design ...

Protalix BioTherapeutics' prGCD Named One of the Five Most Promising Drugs Entering Phase III Trials by Thomson Scientific

...which is used as an enzyme replacement therapy for gaucher disease, was named one of the Five Most Promising ... the commercialization of prGCD as a treatment for gaucher disease patients. About Protalix BioTherapeutics...ate, prGCD, for its enzyme replacement therapy for gaucher disease, a lysosomal storage disorder in humans, a...

One shot of gene therapy spreads through brain in animal study

...her examples of lysosomal storage diseases are Tay-Sachs disease, Hunter disease and Pompe disease. In some types of the lysosomal storage disorder gaucher disease, physicians can supply the missing enzyme to patients and successfully relieve disease symptoms. However, for Sly syndrome and most other lyso...

Russell Investment Group Advises Protalix Biotherapeutics of Index Adjustment

...d enrollment and treatment of patients in its pivotal phase III clinical trial of its lead product candidate, prGCD, for enzyme replacement therapy of gaucher disease, a lysosomal storage disorder in humans. The final design of the pivotal phase III clinical trial is based on an agreement reached by Protalix...
Gaucher in Medical Technology

Shire Reports Positive Results From First of Three Phase III Trials of velaglucerase alfa for Type 1 Gaucher Disease and Provides Important Updates on Interactions With FDA

LEXINGTON, Massachusetts, August 3 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY ), the global specialty biopharmaceutical company, today reported positive results from the first of three Phase III studies of velaglucerase alfa, its enzyme replacement therapy in development fo...

Shire Receives Fast Track Designation for velaglucerase alfa for Gaucher Disease

LEXINGTON, Massachusetts, July 16 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY ), the global specialty biopharmaceutical company, announces it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for velaglucerase alfa, its enzyme replacement t...

Shire has Filed a Treatment Protocol for Velaglucerase Alfa for Gaucher Disease

LEXINGTON, Massachusetts, July 6 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY ), the global specialty biopharmaceutical company, announces that, at the request of the FDA, in view of a potential restriction on the availability of the current approved and marketed treatment fo...

Amicus Therapeutics Presents Preclinical Data from Studies of Plicera for Gaucher Disease

Studies Show Increased Enzyme Levels in Two Most Common Mutations CRANBURY, N.J., March 20, 2007 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announ...

Amicus Therapeutics Presents Positive Results From Phase 1 Clinical Studies of Plicera for Gaucher Disease

Phase 2 Clinical Trials Initiated CRANBURY, N.J., March 21, 2007 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announced today that it will present p...

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

...gostat tartrate), and its use for the treatment of gaucher disease. The patent will expire in 2027. J...our protection in the United States for Plicera in gaucher and is one example of our ongoing efforts to estab...efficacy, as measured by the standard endpoints in gaucher disease. Amicus previously reported that enrollme...

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

...t hydrochloride) for Fabry disease and Plicera(TM) (afegostat tartrate) for gaucher disease. About the Phase 2 Trial with AT2220 Amicus initiated a m...e and is conducting Phase 2 clinical trials of Plicera for the treatment of gaucher disease and AT2220 for the treatment of Pompe disease. Forward-Lookin...

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

... (TM) (isofagomine tartrate) for the treatment of gaucher Disease A Phase 2 clinical trial of Plicera in gaucher patients is ongoing. This 6 month study is designe... efficacy as measured by the standard endpoints in gaucher disease. Target enrollment of 16 patients for this...

Amicus Therapeutics Announces Second Quarter 2008 Financial Results

...gulatory plan in Fabry as well as executing on our gaucher and Pompe Phase 2 clinical programs, and we have t...macology studies to support the Phase 3 program. gaucher Disease: Plicera(TM) (isofagomine tartrate) is a...peutic in Phase 2 development for the treatment of gaucher disease. As previously disclosed, Amicus amended t...

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

...ted Phase 2 clinical trials of Amigal for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera(TM) for the treatment of gaucher disease and AT2220 for the treatment of Pompe disease. Forward-Looking Statements This press release contains "forward-looking statements" within ...
Gaucher in Medical Dictionary

Swallowing difficulty

...lude: A ... Conditions causing symptom Swallowing difficulty including possible medical causes, diseases, disorders, and related symptoms. gaucher disease overview ... Another difficulty can occur when weak throat muscles cannot move all of the ... Avoid talking while eating/ swallowing ... ...
Gaucher in Biological News

UC and partners awarded $23 million to transform discoveries into real-world health solutions

...many research successes. UC faculty and Cincinnati Children's and VA researchers working in the GCRC have been at the forefront of Reye's Syndrome and gaucher disease, and have used novel medications to treat rare diseases such as lymphangioleiomyomatosis (LAM) and Fanconi anemia. "Successes already reali...

The American Society of Human Genetics hosts 58th Annual Meeting in Philadelphia

...cluding: the results of a genome-wide association scan for genetic determinants of warfarin dosage, a successful enzyme replacement therapy for Type I gaucher Disease, and breakthrough research on a new treatment that has the potential to be the first successful therapy for Huntington's Disease. OTHER ASH...

Scientists rebuild ancient proteins to reveal primordial Earth's temperature

... from biology and geology show that Earths environment has continuously been changing since life began, and life has adapted appropriately to survive, gaucher said. ...

Male contraception: One door opens, another closes

...roduction, Oct. 25). The drug is already FDA-approved and on the market in both Europe and the United States for treatment of a rare genetic disorder, gaucher disease. So scientists were very excited when they realized that it was acting as a contraceptive in their male mice at a tenth of the disease-treatme...
Gaucher in Biological Technology

Amicus Therapeutics Presents Positive Data from Phase 2 Clinical Trial of Plicera(TM) for Gaucher Disease

Data Support Commencement of Longer-term Phase 2 Switching Study CRANBURY, N.J., March 13 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small-molecule, orally administered pharmacological chaperones for the treatment of human genetic diseases, announced today ...

Amicus Therapeutics Announces Second Quarter 2009 Financial Results

...trial. Plicera (TM) (afegostat tartrate) for the treatment of gaucher Disease A Phase 2 clinical trial of the Company's investigational d... dosing, and preliminary efficacy, as measured by the standard endpoints in gaucher disease. The Company expects to report the results early in the fourth qua...

Shire plc: Core Portfolio of Products Delivers 20% Sales Growth

... results from our trial of velaglucerase in naïve gaucher patients. A treatment protocol for early access ha...on. Velaglucerase alfa - for the treatment of gaucher disease - On July 16, 2009 Shire announced th... therapy in development for the treatment of gaucher disease. On July 30 Shire began the rolling ...

Shire begins the year with a strong performance

...the fourth quarter of 2009; - DAYTRANA for the treatment of ADHD in adolescents in the US in 2010; - Velaglucerase Alfa for the treatment of gaucher disease in the US and the EU in 2010; and - VYVANSE for the treatment of ADHD, in ex-US and ex-EU regions starting in 2010, and in th...

Publication of Shire plc's Annual Report 2008

... designed to treat such debilitating conditions as gaucher disease, Fabry disease and Metachromatic Leukodyst...Velaglucerase alfa (GA-GCB) - for the treatment of gaucher disease Velaglucerase alfa is an enzyme rep...ement therapy being developed for the treatment of gaucher disease. Shire has completed enrolment in a worldw...

Shire Delivers Excellent Growth for the Year, With the new Product Portfolio Achieving Sales of $1 Billion

...dexamfetamine dimesylate) for use in the treatment of ADHD in children in Canada during 2010; and - Velaglucerase alfa for the treatment of gaucher disease in the US and the EU in 2010. CASHFLOW AND LIQUIDITY Shire's robust balance sheet includes $218 million of cash and cash...

Amicus Therapeutics Announces Change to Board of Directors

...mpleted Phase 2 clinical trials of Amigal for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera for the treatment of gaucher disease and AT2220 for the treatment of Pompe disease. FOLD-G ...

Amicus Therapeutics Announces Fourth Quarter and Full Year 2008 Financial Results

... our lead clinical development programs for Fabry, gaucher and Pompe diseases while maintaining a strong fina...(TM) (isofagomine tartrate) for the treatment of gaucher Disease During the fourth quarter, Amicus al...g its ongoing Phase 2 clinical trial of Plicera in gaucher disease. This 6-month study is designed to evaluat...

Amicus Therapeutics Announces Fourth Quarter and Year End 2008 Financial Results Release Date

...mpleted Phase 2 clinical trials of Amigal for the treatment of Fabry disease and is conducting Phase 2 clinical trials of Plicera for the treatment of gaucher disease and AT2220 for the treatment of Pompe disease. FOLD -G ...

Amicus Therapeutics Adds Sol J. Barer, Ph.D. to Board of Directors

...echnology and continue to advance our three lead drug candidates for Fabry, gaucher and Pompe diseases. We look forward to Dr. Barer's leadership and guidance...e and is conducting Phase 2 clinical trials of Plicera for the treatment of gaucher disease and AT2220 for the treatment of Pompe disease. FOLD-G ...
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