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Pompe in Medical News

Cure For Pompe Disease

Pompe disease is a rare inherited enzyme deficiency that destroys muscles and can kill infants at a very young age. Myozyme is a drug developed by Genzyme// to treat this disease. Genzyme, a biotechnology company in Cambridge, Mass said that it has spent more than $500 million since 1998 to develop...

BioMarin Announces Second Quarter 2009 Financial Results

...hich may upregulate utrophin, a potential replacement for the missing dystrophin protein in DMD patients. BMN-103 - alpha-glucosidase (GAA) for pompe Disease: BMN-103 is a highly phosphorylated GAA enzyme, which the company believes could result in more efficient uptake in cells and potentially lea...

Genzyme Delivers Strong Fourth Quarter to Conclude Outstanding Year

... Late-Onset Treatment Study of Myozyme met its co-primary endpoints, confirming the benefit of the product for patients across the spectrum of pompe disease. The company has begun submitting the results of this study for presentation at medical meetings and will pursue the inclusion of the...

One shot of gene therapy spreads through brain in animal study

...es, and the waste deposits interfere with basic cell functions. Other 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 rel...

New DNA Tests Advances Treatment of Lysosomal Storage Diseases

... - Maroteaux-Lamy), and MPS I (Hurler, Hurler-Scheie, or Scheie syndrome). ERT also is in development for other lysosomal storage conditions including pompe (also known as glycogen storage disease type II), MPS II (Hunter syndrome), Niemann-Pick Disease, and MPS IV (Morquio syndrome). ...

Gene Therapy Technique for Inherited Heart Problems in The Pipeline

...begin as soon as early next year for patients with pompe disease, a rare form of muscular dystrophy that is...t pumps out an enzyme missing in a mouse model for pompe disease. The ailment is caused by a single defecti...esearchers hope to launch a trial in patients with pompe disease early next year. ‘Many of the inborn error...
Pompe in Medical Technology

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

CRANBURY, N.J., Feb. 27 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD ) announced today that the Company has suspended enrollment for the Phase 2 clinical trial of its investigational drug AT2220 (1-deoxynojirimycin HCI) for the treatment of Pompe Disease and that it has received v...

Amicus Therapeutics Begins Phase 2 Clinical Trial of AT2220 in Pompe Disease

CRANBURY, N.J., June 3 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD ), a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, today announced that it has initiated a Phase 2 clinical tri...

Amicus Therapeutics Announces Positive Results From Two Phase 1 Clinical Studies of AT2220 for Pompe Disease

CRANBURY, N.J., Oct. 23 /PRNewswire-FirstCall/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of a range of human genetic diseases, announced today positive results from two recently completed Phas...

Amicus Therapeutics Announces Plans to Present Phase 1 Data for AT2220 for Pompe Disease

CRANBURY, N.J., Sept. 18 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD ) today announced that it plans to present the results of Phase 1 studies of AT2220 (1-deoxynojirimycin HCl) for Pompe Disease at the American Society of Human Genetics (ASHG) Annual Meeting on October 23-27 ...

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

...2220 (1-deoxynojirimycin HCl) for the treatment of pompe Disease A Phase 2 clinical trial of AT2220 in adult pompe patients is ongoing. The trial includes an 11 week...ility of a combination that may be appropriate for pompe patients who are not amenable to chaperone monothe...

Amicus Therapeutics Announces Second Quarter 2008 Financial Results

...ogram and Initiation of Phase 2 Clinical Trial in pompe are Highlights for the ...ted a Phase 2 trial of AT2220 for the treatment of pompe disease, which is the third product from our pharm...cokinetics study is currently under development. pompe Disease: AT2220 (1-deoxynojirimycin HCl) is an i...

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

...eatment 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 the meaning of the Private Securities Litigati...

Amicus Therapeutics Presents Data from Clinical Ex Vivo Response Study and Phase 1 Studies of AT2220

... Data Suggest Majority of pompe Patients May Be Amenable to Chaperone ...icus' compound in development for the treatment of pompe disease, at the American College of Medical Geneti...y designed to test the effect of AT2220 on various pompe mutations. Blood and skin samples were collected f...

Amicus Therapeutics to Present at the 26th Annual JPMorgan Healthcare Conference

... clinical trials of Plicera(TM) for the treatment of Gaucher disease. The Company has completed Phase 1 clinical trials of AT2220 for the treatment of pompe disease. Forward-Looking Statements Amicus cautions you that statements included in this press release that are not a description of historical fa...

One Shot of Gene Therapy Spreads Through Brain in Animal Study

...es, and the waste deposits interfere with basic cell functions. Other 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 relie...
Pompe in Biological News

'Show me the money!' MDA greenlights grants

... disease, and spinal muscular atrophy. It also served as the basis for a lifesaving therapy developed by Genzyme for a metabolic muscle disease called pompe disease. At Columbia University, Schon has been awarded a three-year $400,000 grant to continue research into diseases of the mitochondria cause...

Climate change threatens 1 in 5 plant species

...l level in order to preserve broad biodiversity in plant species. Sven pompe and his colleagues from UFZ evaluated the potential impact of climate chang...ecies could lose their niches in habitats such as mountains or moors," Sven pompe from UFZ explains. Migrating species from southern Europe could not compens...

UF scientists test improved gene therapy method for hereditary heart conditions

...begin as soon as early next year for patients with pompe disease, a rare form of muscular dystrophy that is...t pumps out an enzyme missing in a mouse model for pompe disease. The ailment is caused by a single defecti...esearchers hope to launch a trial in patients with pompe disease early next year. "Many of the inborn error...

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

...at accounts for a significant portion of the instances of mental retardation in children. Other examples include Tay-Sachs disease, Hunter disease and pompe disease. In a lysosomal storage disease, cellular debris accumulates within storage areas of cells called lysosomes. In the case of AMD, children are ...

Muscle-targeted gene therapy reverses rare muscular dystrophy in mice

...s born with the inherited muscular disorder called pompe disease usually die before they reach the age of t...eating other muscular dystrophies. Patients with pompe disease have a defect in a key enzyme that convert...onsidered as a potential gene therapy strategy for pompe disease patients, the researchers report in a fort...
Pompe in Biological Technology

Genzyme Study of Myozyme(R) for Late-Onset Pompe Patients Meets Co-Primary Efficacy Endpoints

CAMBRIDGE, Mass., Dec. 13 /PRNewswire-FirstCall/ -- Genzyme Corp. (Nasdaq: GENZ ) announced today that its Late Onset Treatment Study (LOTS) of Myozyme(R) (alglucosidase alfa) met its co-primary efficacy endpoints. The study was undertaken to evaluate the safety and efficacy of Myozyme in juv...

Amicus Therapeutics Announces Second Quarter 2009 Financial Results

...e 2 trial. AT2220 (1-deoxynojirimycin HCl) for the treatment of pompe Disease As previously reported, the Company suspended enrollment fo...se 2 clinical trial of its investigational drug AT2220 for the treatment of pompe Disease and received notice from the U.S. Food and Drug Administration (FDA...

BioMarin Announces First Quarter 2009 Financial Results

...protein in DMD patients. BMN-103 - alpha -glucosidase (GAA) for pompe Disease: BioMarin expects to make a decision on the strategic direction fo...f PEG-PAL Phase II trial 3Q09: Decision on strategy for BMN-103 for pompe Disease 3Q09: Decision on 6R-BH4 cardiovascular program 1Q1...

Shire begins the year with a strong performance

...dose only. The drug was well tolerated. Alba Therapeutics Corporation has a further Phase 2 study ongoing. HGT-3510 - for the treatment of pompe Disease - In February 2009, the Phase 2 clinical trial for HGT-3510 initiated by Amicus Therapeutics Inc. ("Amicus") was placed on cl...

Publication of Shire plc's Annual Report 2008

...tside the US. HGT-3510 for the treatment of pompe disease HGT-3510 is an orally-administered,...cal chaperone being developed for the treatment of pompe disease. In June 2008 Amicus initiated Phase 2 cli...erone being jointly developed for the treatment of pompe disease by Shire and Amicus. This trial was placed...

Amicus Therapeutics Announces Change to Board of Directors

...e 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

...inical development programs for Fabry, Gaucher and pompe diseases while maintaining a strong financial posi...2220 (1-deoxynojirimycin HCl) for the treatment of pompe Disease Amicus continued to make progress th... ongoing Phase 2 clinical trial of AT2220 in adult pompe patients. The trial includes an 11-week treatment ...

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

...e 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

...d continue to advance our three lead drug candidates for Fabry, Gaucher and pompe diseases. We look forward to Dr. Barer's leadership and guidance as Amicus...licera for the treatment of Gaucher disease and AT2220 for the treatment of pompe disease. FOLD-G ...

Amicus Therapeutics to Present at Lazard Capital Markets Healthcare Conference

...e 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. Amicus' press releases are available at http://www.amicustherapeutics.com FOLD -G ...
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