Fabry Disease

Type: Keyphrase
Name: Fabry Disease
First reported 19 hours ago - Updated 9 hours ago - 4 reports

Amicus Therapeutics reports positive phase III data from fabry monotherapy study 011

Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, has announced additional positive data from a Phase 3 study (Study 011) of the oral small molecule chaperone migalastat HCl ("migalastat") ... [Published PBR - News - 9 hours ago]
First reported Oct 15 2014 - Updated Oct 16 2014 - 1 reports

AbbVie to reconsider $55-bn Shire deal

The board of US drug firm AbbVie Inc. yesterday said that it plans to reconsider recommending that shareholders vote in favour of its planned $55 billion (£32 billion) takeover of Ireland-based Shire Plc after the US Treasury changed tax rules to prevent ... [Published domain-b - Oct 15 2014]
First reported Oct 09 2014 - Updated Oct 09 2014 - 1 reports

Amicus Therapeutics, Inc. (FOLD): Redmile Group Trims Position and Books Some Profits from Long-Term Investment

Jeremy Green ‘s Redmile Group has also reduced its stake in  Amicus Therapeutics, Inc. (NASDAQ: FOLD ),  after yesterday,  William Leland Edwards ’ Palo Alto Investors reported the sale of nearly a half of its stake in the company . However, Redmile only ... [Published InsiderMonkey.com - Oct 09 2014]
First reported Oct 04 2014 - Updated Oct 04 2014 - 1 reports

LSDSS faces dual challenge of expensive treatment and absence of orphan drug status in India for ERT

Lysosomal Storage Disorder Support Society (LSDSS) is bogged down by the challenge of exorbitant drug costs and absence of orphan drug status in India for Enzyme Replacement Therapy (ERT) which is the only medication to stall the progression of the deadly ... [Published PharmaBiz - Oct 04 2014]
First reported Oct 01 2014 - Updated Oct 01 2014 - 1 reports

Rare Genetic Disease patients conduct protest demonstration against Delay in Setting up Treatment Center

About 50 known patients are suffering from Lysosomal Storage Disorders in the state of KarnatakaBengaluru, 30 th September, 2014: With an aim to seek support from the government of Karnataka a demonstration was held at Freedom Park, for setting up of ... [Published This Week Bangalore - Oct 01 2014]
First reported Oct 01 2014 - Updated Oct 01 2014 - 1 reports

LSD patients protest, demand facilities from govt

Patients with Lysosomal Storage Disorders (LSD), an inherited disorder, staged a silent protest here on Tuesday demanding that the State government set up a special centre at the Indira Gandhi Institute of Child Health, as promised.Prasanna Kumar B Shirol, ... [Published Deccan Herald - Oct 01 2014]
First reported Sep 30 2014 - Updated Sep 30 2014 - 1 reports

Orphan designation: (3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate, for the: Treatment of Fabry disease

First reported Sep 30 2014 - Updated Sep 30 2014 - 1 reports

Amicus Therapeutics to Present at Leerink Partners Rare Disease Roundtable

CRANBURY, N.J., Sept. 30, 2014 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD) today announced that Bradley Campbell, Chief Operating Officer and Chip Baird, Chief Financial Officer, will present a corporate overview at the Leerink Partners Rare ... [Published Industrial Info Financials - Sep 30 2014]
First reported Aug 28 2014 - Updated Aug 28 2014 - 1 reports

Amicus Therapeutics (FOLD) Stock Rises on High Volume

NEW YORK (TheStreet) --aAmicus Therapeuticsa nearly touched a 52-week high of $7.40 on Thursday as the stock continued to rise on high volume. Last week, the companyaannounced its potential Fabry disease treatment drug migalastat met its primary endpoints ... [Published The Street Latest - Aug 28 2014]
First reported Aug 20 2014 - Updated Aug 21 2014 - 2 reports

Why Amicus Therapeutics (FOLD) Stock Hit a One-Year High Today

NEW YORK (TheStreet) --aAmicus Therapeuticsa surged to a 52-week high of $5.47 on Wednesday after the company announced its potential Fabry disease treatment drugamigalastat met its primary endpoints in a late-stage study. The results showed Fabry patients ... [Published The Street Latest - Aug 20 2014]
First reported Aug 15 2014 - Updated Aug 15 2014 - 1 reports

A family’s fight vs rare disease

Ever since he was a young boy, Alexander Baldoza would experience numbness and tingling, crawling sensations—like ants—crawling on both hands and feet. It got much worse when he had a cold or fever.It was only in 2010 and at 42 years old that Alexander ... [Published Philippine Daily Inquirer - Aug 15 2014]
First reported Jun 23 2014 - Updated Jun 23 2014 - 1 reports

Research and Markets: Global Fabry Disease Pipeline Report 2014-2017

DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/xs3qs6/fabry_disease) has announced the addition of the "Fabry Disease Pipeline Highlights - 2014 Update" report to their offering. The latest report Fabry Disease ... [Published Business Wire Health News - Jun 23 2014]

Quotes

Dr. Daniel Bichet, Full Professor and Section Head, Renal Function & Transport Physiology, University of Montreal, said, "Baseline proteinuria levels are among the most predictive indicators of disease prognosis and kidney function decline in Fabry patients. The data presented today show that when comparing patients with similar levels of proteinuria, patients treated with migalastat are more stable in their kidney function versus untreated patients. These results are very encouraging for migalastat as a treatment for Fabry patients with amenable mutations."
...Jay A Barth, Chief Medical Officer of Amicus TherapeuticsAmicus Therapeutics Inc. "The additional data on substrate reduction show that we can accurately identify patients who may benefit from migalastat. We look forward to meeting with regulatory agencies starting this quarter as we work to make migalastat available for all amenable Fabry patients as quickly as possible."
"Accordingly, AbbVie has notified Shire under the Co-operation Agreement that AbbVie's Board of Directors intends to meet to consider whether to withdraw or modify its recommendation. Under the Agreement, AbbVie must provide three business days' notice of any intention to consider a change in recommendation. Accordingly, AbbVie's Board plans to meet on October 20, 2014, unless Shire agrees to waive the notice" AbbVie said in a statement

More Content

All (31) | News (24) | Reports (0) | Blogs (6) | Audio/Video (0) | Fact Sheets (0) | Press Releases (1)
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Amicus Therapeutics reports positive phase III ... [Published PBR - News - 9 hours ago]
Amicus Therapeutics Reports Positive Phase 3 Da... [Published RTTNews.com - 17 hours ago]
Amicus Therapeutics Announces Additional Positi... [Published Virtual Strategy Magazine - 19 hours ago]
Amicus Therapeutics Announces Additional Positi... [Published Market Pulse Navigator - 19 hours ago]
AbbVie to reconsider $55-bn Shire deal [Published domain-b - Oct 15 2014]
Amicus Therapeutics, Inc. (FOLD): Redmile Group... [Published InsiderMonkey.com - Oct 09 2014]
LSDSS faces dual challenge of expensive treatme... [Published PharmaBiz - Oct 04 2014]
Genzyme : Patent Issued for Methods for Introdu... [Published 4 Traders - Oct 03 2014]
Rare Genetic Disease patients conduct protest d... [Published This Week Bangalore - Oct 01 2014]
LSD patients protest, demand facilities from govt [Published Deccan Herald - Oct 01 2014]
Orphan designation: (3S)-1-azabicyclo[2.2.2]oct... [Published Orphan designations - Sep 30 2014]
Amicus Therapeutics to Present at Leerink Partn... [Published Industrial Info Financials - Sep 30 2014]
Genzyme Announces Launch of "Expression of Hope... [Published 4 Traders - Sep 18 2014]
Amicus Therapeutics (FOLD) Stock Rises on High ... [Published The Street Latest - Aug 28 2014]
Karnataka govt yet to disburse Rs.10 cr for ded... [Published PharmaBiz - Aug 23 2014]
Group still hopeful for passage of rare disease... [Published Philippine Daily Inquirer - Aug 22 2014]
Why Amicus Therapeutics (FOLD) Stock Hit a One-... [Published The Street Latest - Aug 20 2014]
Futures Little Changed With Fed Minutes On Tap [Published Clusterstock - Aug 20 2014]
Yes, Call it a Comeback: Amicus Fabry Drug Post... [Published The Street Latest - Aug 20 2014]
A family’s fight vs rare disease [Published Philippine Daily Inquirer - Aug 15 2014]
Agonists of neurotrophin receptors and their us... [Published PharmCast - Aug 14 2014]
14 Insanely Important Biotech/Drug Trading Cata... [Published The Street Latest - Jul 10 2014]
Amicus Therapeutics Analyst Report; Solid Pipel... [Published Financial Services - Jul 07 2014]
Why Amicus Therapeutics (FOLD) Stock Continues ... [Published The Street Latest - Jul 01 2014]
Research and Markets: Global Fabry Disease Pipe... [Published Business Wire Health News - Jun 23 2014]
Research and Markets: Fabry Disease - Pipeline ... [Published Business Wire Health News - Jun 10 2014]
Orphan designation: 1-Deoxygalactonojirimycin h... [Published Orphan designations - May 08 2014]
Why Amicus Therapuetics (FOLD) Stock Is Surging... [Published The Street Latest - Apr 29 2014]
Protalix BioTherapeutics Announces Oral GCD Dat... [Published GlobeNewswire - Feb 12 2014]
Amicus Therapeutics Highlights Data Featured at... [Published GlobeNewswire - Feb 12 2014]
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Blogs

sort by: Date | Relevance
Amicus Therapeutics reports positive phase III ... [Published PBR - News - 9 hours ago]
Amicus Therapeutics (FOLD), a biopharmaceutical company at the forefront of therapies for rare and orphan diseases, has announced additional positive data from a Phase 3 study (Study 011) of the oral small molecule chaperone migalastat HCl ("migalastat") ...
Orphan designation: (3S)-1-azabicyclo[2.2.2]oct... [Published Orphan designations - Sep 30 2014]
Futures Little Changed With Fed Minutes On Tap [Published Clusterstock - Aug 20 2014]
By Chuck Mikolajczak NEW YORK (Reuters) - Stock futures were little changed on Wednesday after the S&P 500 notched two straight days of gains and ahead of the release of minutes from the most recent meeting of the Federal Reserve. Investors will ...
Research and Markets: Global Fabry Disease Pipe... [Published Business Wire Health News - Jun 23 2014]
DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/xs3qs6/fabry_disease) has announced the addition of the "Fabry Disease Pipeline Highlights - 2014 Update" report to their offering. The latest report Fabry Disease ...
Research and Markets: Fabry Disease - Pipeline ... [Published Business Wire Health News - Jun 10 2014]
DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/fhx488/fabry_disease) has announced the addition of the "Fabry Disease - Pipeline Review, H1 2014" report to their offering. This report provides comprehensive information ...
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Press Releases

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Amicus Therapeutics Analyst Report; Solid Pipel... [Published Financial Services - Jul 07 2014]
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