Navigation Links
BioMarin Initiates Phase 2 Study for GALNS in Patients Under Five Years of Age With MPS IVA
Date:11/29/2011

NOVATO, Calif., Nov. 29, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 2 study for GALNS (N-acetylgalactosamine 6-sulfatase) in patients under five years of age with mucopolysaccharidosis IVA (MPS IVA). 

"We expect this study to build our knowledge base in enzyme replacement therapies wherein clinicians believe earlier therapy is more impactful," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin.  "Comparatively speaking, this study is starting years before a similar study for Naglazyme, which was done as a post-marketing commitment.  We will also conduct a study in non-ambulatory patients, which, along with the study in patients under five years of age, will encompass the spectrum of MPS IVA patients not included in the Phase 3 trial.  Enrollment in the Phase 3 pivotal study is on track for results in the second half of 2012, and its execution is BioMarin's highest product development priority."

The primary objective of the Phase 2, open-label, multinational clinical study is to evaluate the safety and tolerability of infusions of GALNS at a dose of 2.0 mg/kg/week over a 52-week period in ten to fifteen MPS IVA patients under five years of age.  The secondary objectives are to evaluate urinary keratan sulfate (KS) levels and growth velocity.

About BioMarinBioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint ve
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2010 PR Newswire.
All rights reserved

Page: 1 2 3

Related medicine technology :

1. BioMarin Announces FDA Approval of Expanded Biologics Manufacturing Facility
2. BioMarin to Host a Research and Development Day on December 8th
3. BioMarin Announces Third Quarter 2011 Financial Results
4. BioMarin to Present at the UBS Global Life Sciences Conference
5. BioMarin to Present at the Morgan Stanley Global Healthcare Conference
6. BioMarin to Present at the Baird Health Care Conference
7. BioMarin to Present at the Wedbush Life Sciences Conference
8. BioMarin Announces Second Quarter 2011 Financial Results
9. BioMarin to Host Second Quarter 2011 Financial Results Conference Call and Webcast on Thursday, July 28 at 5:00 p.m. ET
10. BioMarin Agrees to Acquire Biologics Manufacturing Plant in Ireland from Pfizer
11. BioMarin to Present at the William Blair Growth Stock Conference
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/21/2014)... PARK, N.J. , Aug. 21, 2014 /PRNewswire/ ... pharmacy group purchasing organization (GPO), is pleased to ... to collaborate on a host of innovative specialty ... help Aurora deliver enhanced therapy management services to ... Management (OTM) system. Aurora will use ...
(Date:8/21/2014)... , Aug. 21, 2014 CVS Caremark Corporation (NYSE: ... applicable Reference Yields and consideration payable in connection with its ... August 7, 2014 for (1) any and all of its ... and (2) up to a maximum amount of its 6.125% ... 5.750% Senior Notes due 2017 (collectively, the "Maximum Tender Offer ...
(Date:8/21/2014)... -- Professional Compounding Centers of America (PCCA) will ... three best scientific presentations at the sixth ... Formulating Better Medicines for Children  conference, held ... Greece , includes dozens of poster-style scientific ... three winners will be selected by a panel ...
Breaking Medicine Technology:Aurora Health Care Partners with Armada Health Care to Deliver Added Pharmacy Capabilities to Specialty Patients 2Aurora Health Care Partners with Armada Health Care to Deliver Added Pharmacy Capabilities to Specialty Patients 3CVS Caremark Corporation Announces Early Results of Tender Offers, Pricing, Election of Early Settlement and Increase in the Maximum Tender Offer Amount 2CVS Caremark Corporation Announces Early Results of Tender Offers, Pricing, Election of Early Settlement and Increase in the Maximum Tender Offer Amount 3CVS Caremark Corporation Announces Early Results of Tender Offers, Pricing, Election of Early Settlement and Increase in the Maximum Tender Offer Amount 4CVS Caremark Corporation Announces Early Results of Tender Offers, Pricing, Election of Early Settlement and Increase in the Maximum Tender Offer Amount 5CVS Caremark Corporation Announces Early Results of Tender Offers, Pricing, Election of Early Settlement and Increase in the Maximum Tender Offer Amount 6Professional Compounding Centers of America will award top poster-session winners at European Paediatric Formulation Initiative in Athens 2
... Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX ), ... human disease, announced today that Arthur T. Sands, ... will present at the Stifel Nicolaus Healthcare Conference in ... Eastern Time.  Dr. Sands will provide an overview on ...
... 2011 Reportlinker.com announces that a new market ... Pharmaceutical Distributors - Global ... This register contains information about 2800 ... register includes the following information about the ...
Cached Medicine Technology:Lexicon Pharmaceuticals to Present at Stifel Nicolaus Healthcare Conference 2
(Date:8/21/2014)... effective vaccination, polio is considered nearly eradicated. Each year ... scientists of the University of Bonn, together with colleagues ... that was able to resist the vaccine protection to ... outbreak in the Congo in 2010. The pathogen could ... results appear now in the magazine PNAS . ...
(Date:8/21/2014)... Irvine, CA (PRWEB) August 21, 2014 ... co-founder of American Corporate Health, was recently ... most influential professionals in the field of ... the National Workers’ Compensation and Occupational Medicine ... practitioners, including attorneys, physicians, nurses, case managers, ...
(Date:8/21/2014)... NY (PRWEB) August 21, 2014 Hospital ... a “topping off” ceremony for the new building at ... construction phase of the $125 million expansion and modernization ... a major donation from George S. Kaufman, Chairman of ... area to the Ambulatory Care Pavilion will be named. ...
(Date:8/21/2014)... to a new market report published by Transparency Market ... Share, Growth, Trends and Forecast, 2012 - 2018," the ... million in 2011 and is estimated to reach a ... CAGR of 6.3% from 2012 to 2018. , Browse ... , Fundus cameras market growth in recent years has ...
(Date:8/21/2014)... 2014 The new program, WellnessConnect ... Companies and builds on Accountable Health Solutions’ DiabetesConnect? ... combined with Accountable Health’s robust member engagement capabilities, ... and adds a custom incentive platform to help ... provider visits, HbA1c testing and more. ...
Breaking Medicine News(10 mins):Health News:Polio: Mutated virus breaches vaccine protection 2Health News:Dr. Vasili Gatsinaris Named One of the Top 50 Most Influential in Workers’ Compensation 2Health News:“Topping Off” Ceremony Marks Major Milestone in Mount Sinai Queens Construction Project 2Health News:“Topping Off” Ceremony Marks Major Milestone in Mount Sinai Queens Construction Project 3Health News:“Topping Off” Ceremony Marks Major Milestone in Mount Sinai Queens Construction Project 4Health News:Fundus Cameras Market: Product Type- Mydriatic, Non-Mydriatic, Hybrid and ROP Cameras; End Users- Hospitals, Ophthalmology Clinics and Ophthalmic & Optometrist Offices 2Health News:Accountable Health Expands Diabetes Management Solutions to Address Gaps in Provider Care 2Health News:Accountable Health Expands Diabetes Management Solutions to Address Gaps in Provider Care 3
... VANCOUVER, March 17 /PRNewswire-FirstCall/ - QLT Inc. ... Food and Drug Administration (FDA),has completed its review ... and has removed the glucose-6-phosphate dehydrogenase (G6PD),screening and ... to report the FDA decision to change the,Aczone ...
... Program Shows the Need Is Still There, WASHINGTON, ... who are still suffering emotionally from the,effects of the ... The American Red Cross Hurricane Recovery Program (HRP) has,announced ... Program until May 31, 2008. The Red Cross Emotional ...
... Hologic, Inc.,(Nasdaq: HOLX ) today announced that Patrick ... be presenting at the Lehman,Brothers Eleventh Annual Global Healthcare ... Hotel in Miami, Florida., Interested parties are invited ... presentation on Thursday, March 20, at 10:15 a.m.,Eastern Time ...
... The U.S. Pharmacopeial (USP) Convention today announced a ... (USP), an official compendium of the United States. ... and potentially fatal health hazards associated with diethylene ... in formulations of many pharmaceutical syrups and in ...
... staff can now be on the same page with ... Gua Para el Paciente Sobre Procedimientos de la Medicina ... both English and Spanish, the book explains in simple ... can expect prior to and during more than 30 ...
... (NYSE: BMY ) will present at the ... Brian Daniels, M.D., senior vice president,Global Development, will ... p.m. ET today., Bristol-Myers Squibb will also ... Wednesday, March 19, 2008. John Celentano, senior,vice president, ...
Cached Medicine News:Health News:QLT announces positive FDA action on Aczone(TM) 2Health News:QLT announces positive FDA action on Aczone(TM) 3Health News:Red Cross Announces an Extension for Emotional Support Assistance for 2005 Hurricane Survivors 2Health News:Hologic to Present at Lehman Brothers Healthcare Conference 2Health News:USP announces revised glycerin monograph 2Health News:Flip-chart book translates complicated medical procedures into easy-to-understand language 2
... a national reference laboratory and a worldwide ... ARUP offers an extensive test menu of ... clinical and anatomic pathology. Owned by the ... more than half of the nation's university ...
... ARUP Laboratories is a national reference laboratory ... research and development. ARUP offers an extensive ... medical tests in clinical and anatomic pathology. ... Laboratories' clients include more than half of ...
... national reference laboratory and a worldwide leader ... offers an extensive test menu of highly ... and anatomic pathology. Owned by the University ... than half of the nation's university teaching ...
... reference laboratory and a worldwide leader in ... an extensive test menu of highly complex ... anatomic pathology. Owned by the University of ... half of the nation's university teaching hospitals ...
Medicine Products: