Navigation Links
BioMarin Receives Orphan Drug Designation from the FDA for BMN-701 for the Treatment of Pompe Disease
Date:8/30/2010

138,000 births.  The current standard of care is Genzyme's Myozyme and Lumizyme.  Prognosis with standard of care is stabilization of the disease or minor improvements for the majority of adult onset patients.  

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 venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine phosphate), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Other product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in clinical development for the treatment of MPS IVA and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking StatementThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations related to regulatory actions on BMN-701 and the development and efficacy of BMN-701. These forward-looking statements are predictions and involve risks and uncertainties such that
'/>"/>

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

Page: 1 2 3

Related medicine technology :

1. BioMarin Partner AnGes MG, Inc. Submits BLA to Japanese Ministry of Health
2. BioMarin Announces FDA Approval for Kuvan
3. BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU
4. BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease
5. BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome
6. BioMarin Initiates Phase 2 Clinical Study of PEG-PAL in PKU
7. BioMarin Acquires Huxley Pharmaceuticals, Inc.
8. BioMarin to Present at the Oppenheimer Healthcare Conference
9. BioMarin Announces Third Quarter 2009 Financial Results
10. BioMarin to Acquire LEAD Therapeutics
11. BioMarin to Present at the Credit Suisse Healthcare Conference
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:9/1/2015)... Sept. 1, 2015 Sargas Pharmaceutical Adherence ... and Drug Adherence m Health applications along with ... process of non face-to-face monitoring of twenty minutes ... release of our apps, doctors are now well ... with our ONCHIT certified Physician, Pharmacy and Patient ...
(Date:9/1/2015)... Within just two years after reaching a ... reached 3,000 employees worldwide. This increase is reflecting ... drug delivery market and SHL,s effective commitment, productivity ... the design and manufacturing of auto injectors and ... capabilities, a lean supply chain, strong partnerships, and ...
(Date:9/1/2015)... 2015 Egalet Corporation (Nasdaq: EGLT ... focused on discovering, developing, and commercializing innovative pain ... scientific data on SPRIX ® (ketorolac tromethamine) ... product candidate, and Egalet-002, an abuse-deterrent, extended-release, oral ... 2015 to be held September 8 to 12 ...
Breaking Medicine Technology:SPAC International's Drug Adherence and Chronic Care Management App Now available at Apple App and Google Play stores 2SHL Group Reaches Major Milestone of 3000 Employees 2Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 2Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 3Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 4Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 5Egalet Announces Scientific Presentations at PainWeek 2015 Meeting 6
... Inc., a privately-held therapeutic device company, today announced ... with Bezwada Biomedical to develop and commercialize Bezwada,s ... Biomedical is an innovation-driven company with proprietary technology ... polyamides derived from hydrolyzable isocyanates. This technology can ...
... Aug. 31, 2011 Assured Pharmacy, Inc. (Pink Sheets: ... that provide advanced prescription services to a diverse array ... it sold in a private placement to an accredited ... an aggregate principal amount of $400,000 due December 1, ...
Cached Medicine Technology:ORTHOCON Announces Signing of Exclusive, Worldwide License Agreement With Bezwada Biomedical 2Assured Pharmacy, Inc. Receives Gross Proceeds of $400,000 from Sale of Convertible Debentures and Warrants 2Assured Pharmacy, Inc. Receives Gross Proceeds of $400,000 from Sale of Convertible Debentures and Warrants 3
(Date:9/1/2015)... ... September 01, 2015 , ... USA Medical Card, a ... week to coincide with back-to-school and the start of “sick season” for many ... elementary school children are sick an average of 8 to 12 times each ...
(Date:9/1/2015)... ... September 01, 2015 , ... Éminence Organic Skin ... the VitaSkin™ Exfoliating Peels. This revolutionary collection of natural peel solutions complements the ... Clear. , Formulated with alpha hydroxy acids and natural active ingredients, these ...
(Date:9/1/2015)... , ... September 01, 2015 , ... Dr. Trace Curry ... JourneyLite of Cincinnati. In the last month the FDA has approved two intra-gastric ... ™. These procedures represent the first incisionless, reversible, weight loss procedures available to ...
(Date:9/1/2015)... ... September 01, 2015 , ... ... for Alfresco, a next-generation customer communications archiving solution integrated with Alfresco‘s enterprise ... from Crawford and helps organizations save millions of dollars in printing costs ...
(Date:9/1/2015)... ... September 01, 2015 , ... Award-winning ... pick-up artists about their experiences of female beauty, women’s emotions, fertility, female violence ... issues, as well as their relationships with other men, documenting how the power ...
Breaking Medicine News(10 mins):Health News:USA Medical Card Launches New FAQ Video During Back-to-School 2Health News:Éminence Organic Skin Care Introduces Results-Oriented Exfoliating Peels for Every Skin Type 2Health News:Éminence Organic Skin Care Introduces Results-Oriented Exfoliating Peels for Every Skin Type 3Health News:Dr. Trace Curry Performs First ORBERA™ Intra-gastric Balloon in Ohio at JourneyLite, a New Incisionless Option For The Treatment of Obesity 2Health News:Introducing Next-Generation Archiving Solution PRO Archiver for Alfresco® 2Health News:Introducing Next-Generation Archiving Solution PRO Archiver for Alfresco® 3Health News:Female Porn Director Explains Male Insecurities about Women in a New Book 2
... Psoriasis Cure Now has,selected its $7,500 Grand Prize winner ... Monica, California walks away with the big prize,after a ... other,Finalists each will receive $250 in the contest. The ... ., "Brendan Hayward captured, in just 30 ...
... 12 International,Isotopes Inc. (OTC Bulletin Board: INIS) ... months ending September 30, 2007. REVENUE: Revenue ... 2007, was $1,486,112 and $3,592,049 respectively, as compared ... 2006. These numbers,represent an increase of 2% for ...
... health care risk,management and insurance provider FinCor Holdings ... ended Sept. 30, 2007, including significant,increases in net ... of the year. The privately held Lansing, ... risk management products,and services for health care providers ...
... HLM Venture Partners announced today,that it has led ... Inc. CaseNET, based in Waltham, MA, is an early-stage ... government,agencies a software solution for medical and care management. ... to the round and was joined,by existing investors Sigma ...
... 12 The American Medical,Association (AMA) proudly presented ... Service Award Saturday at its semi-annual,policy-making meeting. Dr. ... award, which is granted for meritorious service in ... has devoted himself to advancing medical education," said,AMA ...
... 12 Dileep G. Bal, M.D.,received the 2007 ... AMA Foundation, the award recognizes physicians who have ... activities. The,award was presented to Dr. Bal, a ... during the opening session of the AMA,s semi-annual,policy-making ...
Cached Medicine News:Health News:Psoriasis Cure Now Video Contest $7,500 Grand Prize Winner Announced 2Health News:International Isotopes Inc. Announces Third Quarter and Nine Months 2007 Financial Results 2Health News:International Isotopes Inc. Announces Third Quarter and Nine Months 2007 Financial Results 3Health News:International Isotopes Inc. Announces Third Quarter and Nine Months 2007 Financial Results 4Health News:FinCor Holdings Posts Double-Digit Increases in Revenue and Net Income for Third Quarter 2Health News:FinCor Holdings Posts Double-Digit Increases in Revenue and Net Income for Third Quarter 3Health News:HLM Venture Partners Leads Series B Financing of CaseNET, Inc. 2Health News:Portland Physician Wins AMA Distinguished Service Award 2Health News:AMA Foundation Honors Hawaii Physician 2
... 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 ...
... 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 ...
... 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: