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Fabry Disease

Type: Keyphrase

Name: Fabry Disease

First reported 13 hours ago - Updated 13 hours ago - 1 reports

Amicus Therapeutics' CEO Presents at 2013 UBS Global Healthcare Conference (Transcript)

Amicus Therapeutics, Inc. (FOLD) 2013 UBS Global Healthcare Conference Call May 21, 2013 1:30 PM ETMatthew Roden - UBSGood evening everyone. My name is Matt Roden. I am one of biotech analyst here at UBS and I'd like to thank everyone for attending the ... [Published Seeking Alpha - 13 hours ago]

Entities: Enzyme, Protein, Fabry Disease

First reported May 20 2013 - Updated May 20 2013 - 1 reports

Sangamo BioSciences Inc

Sangamo Biosciences Inc Announces New Data From In Vivo Protein Replacement Platform For Development Of ZFP Therapeutics For Monogenic Diseases

Sangamo Biosciences Inc announced the presentation of new data demonstrating the successful application of Sangamo's In Vivo Protein Replacement Platform (IVPRP) to produce therapeutically relevant levels of Factor VIII in a mouse model. Sangamo has partnered ... [Published Reuters - May 20 2013]

Entities: Sangamo BioSciences Inc, In Vivo, Protein, Therapeutics

First reported May 17 2013 - Updated May 17 2013 - 1 reports

Payer focus in the personalised world of oncology and orphan diseases

Personalised medicine is transforming disease understanding and many traditional diagnoses may be fundamentally revised based on the scientific understanding of disease mechanisms at the molecular, genetic and genomic levelsThe National Human Genome Research ... [Published PMLive - May 17 2013]

Entities: Oncology, treatments, Patient, Philadelphia Chromosome, Iressa

First reported May 15 2013 - Updated May 16 2013 - 2 reports

AMICUS

Amicus Therapeutics: The Good And The Bad

Home » Blogs » Amicus Therapeutics (FOLD) is a biopharmaceutical company that is developing novel, first-in-class treatments for a broad range of human genetic diseases, with a focus on delivering new benefits to individuals with lysosomal storage diseases. ... [Published BioPortfolio - May 16 2013]

Entities: Amicus Therapeutics Inc, Fabry Disease, AMICUS, Placebo

First reported May 14 2013 - Updated May 15 2013 - 1 reports

Amicus Therapeutics Inc

Amicus Therapeutics to Present at UBS Global Healthcare Conference

(GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today announced that John F. Crowley, Chairman and Chief Executive Officer, will present a corporate overview at the 2013 UBS Global Healthcare Conference in New York, NY on Tuesday, May 21, 2013 at ... [Published Nasdaq - May 14 2013]

Entities: Amicus Therapeutics Inc, Healthcare Conference, UBS AG

First reported May 13 2013 - Updated May 13 2013 - 1 reports

Shire plc

Shire's New Rare Disease Deal: Interesting Science Meets Unique Financing

the biotechnology company Nimbus Discovery announced an agreement with the specialty pharmaceutical developer Shire plc (NASDAQ: SHPG) that combines an innovative technology for drug discovery with a novel financing method to move that technology forward. ... [Published Seeking Alpha - May 13 2013]

Entities: Shire plc, Atlas Venture, Drugs, Biotechnology

First reported May 09 2013 - Updated May 10 2013 - 3 reports

Amicus' CEO Discusses Q1 2013 Results - Earnings Call Transcript

Amicus Therapeutics, Inc. (FOLD) Q1 2013 Results Earnings Call May 9, 2013 5:00 PM ETOperatorGood day, ladies and gentlemen. And welcome to the Amicus Therapeutics First Quarter 2013 Results Conference Call. At this time, all participants are in a listen-only ... [Published Seeking Alpha - May 10 2013]

Entities: AMICUS, Fabry Disease, Patient, Placebo, John Crowley

First reported May 09 2013 - Updated May 09 2013 - 1 reports

Shire signs rare disease pill pact with Nimbus

Shire has linked up with the USA's Nimbus Discovery to develop small molecule oral drugs for several rare genetic diseases.The firms say that the goal of the collaboration is to use Nimbus' computational chemistry approach to discover disease-altering ... [Published Pharma Times - May 09 2013]

Entities: Shire plc, Genetic Disorder, ELAPRASE, VPRIV, REPLAGAL

First reported May 09 2013 - Updated May 09 2013 - 1 reports

Lobby group dissappointed at cuts to drugs buying

A Government decision to axe $30 million from its medicine buying budget is being criticised by a lobby group.Medicines New Zealand says the money could have allowed Pharmac to invest in new drugs, and help patients with rare diseases.Currently people ... [Published ONE News - May 09 2013]

Entities: Drugs, Heather Roy, Brenda Jones

First reported May 02 2013 - Updated May 03 2013 - 3 reports

Shire shares in unexpected fall

The drugs manufacturer suffered an unexpected drop in shares Sales of Dermagraft, a treatment for diabetes-related ulcers, dropped 62 per cent. The stock fell 135p to 1885p as Shire predicted a high single-digit rise in sales, rather than the low double-digit ... [Published Daily Express - May 03 2013]

Entities: Shire plc, DERMAGRAFT, REPLAGAL, Fabry Disease

First reported May 02 2013 - Updated May 03 2013 - 2 reports

Sanofi predicts return to growth in second half

But first quarter net income drops by a thirdSolid results from new products and emerging markets helped offset generic competition for Sanofi in the first quarter, with overall sales down just 2.8 per cent in the quarter.Sanofi is still weathering the ... [Published PMLive - May 02 2013]

Entities: Sanofi SA, Aubagio, Fabrazyme, Cerezyme

First reported May 02 2013 - Updated May 02 2013 - 2 reports

Amicus Therapeutics to Announce First Quarter 2013 Financial Results on May 9, 2013

CRANBURY, N.J., (GLOBE NEWSWIRE) -- Amicus Therapeutics () today announced a conference call and live audio webcast on Thursday, May 9, 2013 at 5:00 p.m. ET to discuss financial results for the first quarter ended March 31, 2013.The call with be hosted ... [Published OSIX News - May 02 2013]

Entities: Amicus Therapeutics Inc, AMICUS, Cranbury (United States), Fabry Disease, lysosome

Quotes

More From BioPortfolio on "Renal Ventures Management Announces New President and CEO of its Infusion Subsidiary"

Renal Ventures Management Announces New President and CEO of its Infusion Subsidiary [Published BioPortfolio - May 20 2013]

...of hemophilia A and B, as well as four enzymes absent in a panel of LSDs by Sangamo's collaborators from the laboratory of Katherine A High "The ASGCT meeting provides a great forum to highlight our latest data demonstrating the potential of our ZFP platform to provide novel therapeutic solutions to a broad range of diseases" said Edward Lanphier

Sangamo BioSciences Presents New Data From In Vivo Protein Replacement Platform For Development O ... [Published ADVFN UK - May 20 2013]

The National Human Genome Research Institute defines personalised medicine as "an emerging practice of medicine that uses an individual's genetic profile to guide decisions made in regard to the prevention, diagnosis, and treatment"

Payer focus in the personalised world of oncology and orphan diseases [Published PMLive - May 17 2013]

...John F Crowley, Chairman and Chief Executive Officer of stated, "Amicus had a productive first quarter that was largely focused on the execution of clinical and regulatory activities for monotherapy for Fabry disease, our upcoming clinical studies in Fabry and Pompe, and the build out of our CHART platform. A Type C meeting with the FDA will take place during the second quarter to discuss the statistical analysis plan for while the 12-month data are still blinded. We remain on track to unblind and announce these 12-month results in the third quarter. We are also finalizing the design of our repeat-dose Phase 2b co-administration study for Pompe disease, which we expect to begin in the third quarter. Our strong financial position will continue to support the advancement of these programs as we approach several key milestones throughout the remainder of 2013."

Amicus Therapeutics Announces First Quarter 2013 Financial Results and Corporate Updates GlobeNew ... [Published Yahoo! Finance - May 09 2013]

More Content

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Amicus Therapeutics' CEO Presents at 2013 UBS G... [Published Seeking Alpha - 13 hours ago]
Sangamo Biosciences Inc Announces New Data From... [Published Reuters - May 20 2013]
Renal Ventures Management Announces New Preside... [Published BioPortfolio - May 20 2013]
Sangamo BioSciences Presents New Data From In V... [Published ADVFN UK - May 20 2013]
Sangamo BioSciences Presents New Data From In V... [Published TickerTech.com - May 20 2013]
Payer focus in the personalised world of oncolo... [Published PMLive - May 17 2013]
Amicus Therapeutics: The Good And The Bad [Published BioPortfolio - May 16 2013]
Amicus Therapeutics: The Good And The Bad [Published Seeking Alpha - May 15 2013]
Amicus Therapeutics to Present at UBS Global He... [Published Nasdaq - May 14 2013]
Shire's New Rare Disease Deal: Interesting Scie... [Published Seeking Alpha - May 13 2013]
Amicus' CEO Discusses Q1 2013 Results - Earning... [Published Seeking Alpha - May 10 2013]
Amicus Therapeutics Announces First Quarter 201... [Published Yahoo! Finance - May 09 2013]
Amicus Therapeutics Announces First Quarter 201... [Published GlobeNewswire - May 09 2013]
Shire signs rare disease pill pact with Nimbus [Published Pharma Times - May 09 2013]
Lobby group dissappointed at cuts to drugs buying [Published ONE News - May 09 2013]
Protalix BioTherapeutics to Present at the Oppe... [Published Morningstar.com - May 08 2013]
Protalix BioTherapeutics to Present at the Oppe... [Published Street Insider - May 08 2013]
HA, CPSS, EDGW, SNY, ASTM, UACL Are Seasonally ... [Published TradingCharts.com - May 07 2013]
The Expert View: RBS, Gulf Keystone Petroleum a... [Published Citywire - May 06 2013]
Investing Ideas: Biodefense Stocks to Watch [Published InvestorIdeas.com - May 03 2013]
Shire Beats Earnings in 1Q - Analyst Blog [Published Quote.com United States - May 03 2013]
Shire misses Q1 targets as Replagal sales fall [Published PMLive - May 03 2013]
New Shire chief sets out strategy as sales disa... [Published Pharma Times - May 03 2013]
Shire shares in unexpected fall [Published Daily Express - May 03 2013]
Shire slumps after lowering revenue outlook [Published CNBC - May 02 2013]
Amicus Therapeutics to Announce First Quarter 2... [Published Equities.com - May 02 2013]
Amicus Therapeutics to Announce First Quarter 2... [Published OSIX News - May 02 2013]
Amicus Therapeutics to Announce First Quarter 2... [Published Stockwatch - May 02 2013]
Shire earnings jump but sales disappoint [Published BullBearings - May 02 2013]
UPDATE 1-Shire cuts sales forecast as some drug... [Published Reuters - May 02 2013]

Blogs

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4 Healthcare Stock Stories Starting Off the Sho... [Published Wall St. Cheat Sheet - Feb 19 2013] Novartis ( NYSE:NVS ): Closing price $69.20 Chairman Daniel Vasella will receive a package that might reach $78 million upon his exit, if he abides by a non-compete agreement that disallows him from working for Novartis’s rivals. Via an emailed ...
Amicus & GlaxoSmithKline Report Study 011 Resul... [Published Wall St. Cheat Sheet - Dec 19 2012] On Wednesday, Amicus Therapeutics ( NASDAQ:FOLD ) and GlaxoSmithKline ( NYSE:GSK ) reported the 6-month primary treatment period results from the first Phase 3 global registration study (Study 011) of investigational oral migalastat HCl monotherapy ...
Pfizer & Bristol-Myers Squibb’s Eliquis Success... [Published Wall St. Cheat Sheet - Dec 10 2012] A study published in Saturday’s New England Journal of Medicine showed that Pfizer ( NYSE:PFE ) and Bristol-Myers Squibb Company’s ( NYSE:BMY ) experimental drug Eliquis both prevented blood clots in the legs and lungs and kept them from recurring. Patients ...

Press Releases

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Genzyme Convenes 13th European Fabry Disease Ro... [Published Business Wire Health News - Apr 18 2013]
Research and Markets: Fabry Disease Global Clin... [Published Business Wire Health News - Mar 05 2013]
Amicus Therapeutics Presents Additional Results... [Published GlobeNewswire: Acquisitions News - Feb 13 2013]
Research and Markets: Fabry Disease - Pipeline ... [Published Business Wire Health News - Feb 05 2013]
Protalix BioTherapeutics Announces First Patien... [Published GlobeNewswire: Acquisitions News - Dec 10 2012]