Navigation Links
BioMarin Initiates Phase 1/2 Clinical Trial for GALNS for Morquio A Syndrome
Date:4/21/2009

NOVATO, Calif., April 21 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today the initiation of a Phase 1/2 clinical trial for BMN-110 or N-acetylgalactosamine 6-sulfatase (GALNS), intended for the treatment of the lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), or Morquio A Syndrome. The company expects to report initial results in the first half of 2010.

"We plan to leverage our clinical, manufacturing and regulatory expertise to bring a new therapeutic option to the significant number of untreated Morquio patients around the world," said Henry Fuchs, M.D., Chief Medical Officer of BioMarin. "GALNS has been shown in mice to reach important tissues including cartilage and different zones of the bone such as bone marrow, calcified bone and importantly, the growth plate. Our experiments have also shown that GALNS is taken up into human Morquio chondrocytes in vitro and reaches the lysosome to clear keratan sulfate."

Chris Hendriksz, M.D., Consultant in Metabolic Disorders, Birmingham Children's Hospital, added, "This is a very exciting announcement for our patients who have been waiting so long for a potential therapy. Hope of a therapeutic is now within reach for these patients, and Birmingham Children's Hospital is very honoured to be part of this exciting development."

The Phase 1/2 study is designed as an open-label, within-patient dose escalation trial in approximately 20 patients followed by a treatment continuation phase. All patients to be enrolled in the study have already been identified. During the dose escalation phase of the study, subjects will receive weekly intravenous infusions of BMN-110 in three consecutive 12-week dosing intervals. The objectives of the Phase 1/2 study will be to evaluate safety, pharmacokinetics, pharmacodynamics and
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2009 PR Newswire.
All rights reserved

Page: 1 2 3 4

Related medicine technology :

1. BioMarin Announces Positive Results From Phase 2A Clinical Study of 6R-BH4 in Sickle Cell Disease
2. BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU
3. BioMarin Announces FDA Approval for Kuvan
4. BioMarin Partner AnGes MG, Inc. Submits BLA to Japanese Ministry of Health
5. Dendreon Initiates Phase 1 Clinical Trial of Small Molecule Targeting TRPM8 in Cancer
6. VentiRx Pharmaceuticals Initiates Phase I Clinical Trial of VTX-1463, a Novel TLR8 Agonist for the Treatment of Allergic Rhinitis
7. Osprey Pharmaceuticals U.S.A., Inc. Initiates Phase Ib Clinical Trial in IgA Nephropathy
8. Pharmacyclics Initiates Phase 1 Clinical Trial of Novel Oral Btk Inhibitor for Refractory B-cell Non-Hodgkins Lymphoma
9. Pharmasset Initiates First Time in Human Study of PSI-7851 for the Treatment of Hepatitis C (HCV)
10. Radius Initiates Phase 2a Clinical Trial of RAD1901 in Menopausal Hot Flashes
11. Healthpoint Initiates Phase II, Dose-Finding Study to Evaluate Effectiveness of a Unique Cell Based Therapy in Venous Leg Ulcers
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/26/2014)... 2014  Thoratec Corporation (NASDAQ: THOR ), ... to save, support and restore failing hearts, said today ... On Tuesday, December 2, the company will ... Healthcare Conference.  D. Keith Grossman , President and ... company beginning at 11:30 a.m., Eastern Standard Time (8:30 ...
(Date:11/26/2014)... Nov. 26, 2014  Aethlon Medical, Inc. (OTCQB:AEMD), announced ... to raise approximately $3.3 million at a price of ... one institutional healthcare focused investor. Each unit consists of one ... share purchase warrant. Each whole warrant entitles the holder ... $0.30 for a period of five years from closing.  ...
(Date:11/26/2014)... , Nov. 26, 2014  Cohen, Placitella ... behalf of investors who purchased Nymox Pharmaceutical Corporation ... during the period from January 31, 2011 through ... whether Nymox and certain of its officers and ... in violation of Sections 10(b) and 20(a) of ...
Breaking Medicine Technology:Thoratec Announces Presentations At Investor Conferences For December 2014 2Aethlon Medical Announces $3.3 Million Equity Investment 2Aethlon Medical Announces $3.3 Million Equity Investment 3Cohen, Placitella & Roth, PC Investigating Nymox Pharmaceutical Corporation for Failing to Disclose Material Information 2Cohen, Placitella & Roth, PC Investigating Nymox Pharmaceutical Corporation for Failing to Disclose Material Information 3
(Date:11/27/2014)... (PRWEB) November 27, 2014 Two international ... implants procedure will help one person with significant ... Dr. Saj Jivraj and Dr. Mamaly Reshad of the ... Calif., have launched a website devoted to helping dentist ... of the website would be devoted to helping a ...
(Date:11/27/2014)... “LAB.C” was featured on NewsWatch as part of its monthly ... available to consumers. Mallory Sofastaii, a technology expert and reporter ... with viewers the company offers a cable case, iBed, and ... portability and functionality. A unique mobile company at IFA ... 5S has a portable USB charger built in. The certified ...
(Date:11/27/2014)... This is a professional and in ... an overview analysis, the report introduces Insulin basic ... industry overview, policy analysis, and news analysis, etc. ... information on policy, plans for Insulin Market product ... more. , This report also provides ...
(Date:11/27/2014)... to help with chores while they are home from school, ... self-esteem, and above all, helps parents maintain a somewhat tidy ... do you encourage your children to want to help? Is ... Development Journal, there is. The study found that adults’ language ... lend a hand. , The study found that using ...
(Date:11/27/2014)... November 27, 2014 RBSEO Services, ... search engine optimization service just at $99. RBSEO ... start-ups cannot afford costly search engine optimization service. ... keeping their affordablity and requirements into consideration. ... but it has not compromised with the service ...
Breaking Medicine News(10 mins):Health News:World-Renowned Oxnard All-on-4™ Dental Implants Experts to Give Away Full Mouth Rehabilitation Annually 2Health News:World-Renowned Oxnard All-on-4™ Dental Implants Experts to Give Away Full Mouth Rehabilitation Annually 3Health News:World-Renowned Oxnard All-on-4™ Dental Implants Experts to Give Away Full Mouth Rehabilitation Annually 4Health News:World-Renowned Oxnard All-on-4™ Dental Implants Experts to Give Away Full Mouth Rehabilitation Annually 5Health News:Top Mobile Friendly Accessories were Featured on NewsWatch Television on October 30, 2014 2Health News:Insulin Industry Analysis (Supply, Consumption) for China, America, Korea, France & Japan Research Now at ReportsnReports 2Health News:Insulin Industry Analysis (Supply, Consumption) for China, America, Korea, France & Japan Research Now at ReportsnReports 3Health News:RBSEO Services Announces SEO Services at $99 Only 2
... Brings Strong Industry and Executive Level Experience to CompanyFT. ... (OTC Bulletin Board: NGNM), a leading provider of cancer ... 53 years old, has joined the Company,s Board of ... Executive Officer. The Company also reported that Mr. ...
... Unique Ability of Masimo PVI(TM) to Reflect Acute ... Anesthesia Research Society 83rd Scientific Congress IRVINE, Calif., ... ), the inventor of Pulse CO-Oximetry and Measure-Through ... a new clinical study, independently conducted by researchers ...
... Navigate the Safest, Most Fuel-Efficient Route and Protect Sensitive Medical ... the World,s PoorSUNNYVALE, Calif., March 16 ... Ships , the leader in using hospital ships to deliver ... the latest version of AWT,s BonVoyage System (BVS) to help ...
... greener earth day resolution in 2009? Join media mogul Sally ... expert Lisa Beres as they take listeners on a journey ... March 16 When you set out to work on ... or the upstairs closet, digging through an avalanche of junk ...
... Environmental FootprintARLINGTON, Va., March 16 Practice ... & Johnson to launch a sustainability research ... care sector. The partnership aims to ... care facilities to reduce environmental impacts and ...
... Deerfield ManagementBEDFORD, Mass, March 16 Insulet Corporation ... tubing-free insulin pump technology with its OmniPod(R) Insulin ... into an agreement with leading healthcare investor, Deerfield ... $60 million in financing through a flexible credit ...
Cached Medicine News:Health News:NeoGenomics Appoints Douglas M. VanOort as Executive Chairman and Interim Chief Executive Officer 2Health News:NeoGenomics Appoints Douglas M. VanOort as Executive Chairman and Interim Chief Executive Officer 3Health News:NeoGenomics Appoints Douglas M. VanOort as Executive Chairman and Interim Chief Executive Officer 4Health News:New Clinical Study Finds Masimo Rainbow SET(R) Pulse CO-Oximetry(TM) Accurate in the Noninvasive Monitoring of Fluid Status During Surgery 2Health News:New Clinical Study Finds Masimo Rainbow SET(R) Pulse CO-Oximetry(TM) Accurate in the Noninvasive Monitoring of Fluid Status During Surgery 3Health News:New Clinical Study Finds Masimo Rainbow SET(R) Pulse CO-Oximetry(TM) Accurate in the Noninvasive Monitoring of Fluid Status During Surgery 4Health News:New Clinical Study Finds Masimo Rainbow SET(R) Pulse CO-Oximetry(TM) Accurate in the Noninvasive Monitoring of Fluid Status During Surgery 5Health News:New Clinical Study Finds Masimo Rainbow SET(R) Pulse CO-Oximetry(TM) Accurate in the Noninvasive Monitoring of Fluid Status During Surgery 6Health News:Applied Weather Technology Guides Mercy Ships With Latest BVS Marine Voyage Optimization Software 2Health News:Applied Weather Technology Guides Mercy Ships With Latest BVS Marine Voyage Optimization Software 3Health News:Applied Weather Technology Guides Mercy Ships With Latest BVS Marine Voyage Optimization Software 4Health News:Sally Jessy Raphael Bids Farewell to Her Infamous Red Glasses, Reports Green Nest 2Health News:Practice Greenhealth Enters Partnership with Johnson & Johnson for Health Care Sustainability Research and Training 2Health News:Insulet Receives $60 Million Funding Commitment 2Health News:Insulet Receives $60 Million Funding Commitment 3Health News:Insulet Receives $60 Million Funding Commitment 4Health News:Insulet Receives $60 Million Funding Commitment 5
... is a national reference laboratory and a ... development. ARUP offers an extensive test menu ... in clinical and anatomic pathology. Owned by ... include more than half of the nation's ...
... 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 ...
... 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 ...
Medicine Products: