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:7/10/2014)... CARLSBAD, Calif. , July 10, 2014 /PRNewswire/ ... products and services, has launched the FreeRunner™ knee ... runners, who suffer from patella mal-tracking return to ... anatomy differently than other braces, providing support when ... move normally when they don,t. Patellofemoral ...
(Date:7/10/2014)...  RESMED INC. (NYSE: RMD ) today announced ... ended June 30, 2014 results on Thursday, July 31, 2014, ... release with ResMed,s results will be issued after 1:00 p.m. ... to discuss operating results and future outlook. The ... Pacific Time and the live webcast of the call can ...
(Date:7/10/2014)... , July 10, 2014  BC Technical, the ... MiE America , Inc., a leading provider of ... into a long-term agreement naming BC Technical as the ... MiE and BC Technical announced today ... to establish BC Technical as the service and systems ...
Breaking Medicine Technology:Breg Introduces Knee Brace to Help Runners with Patellofemoral Pain Syndrome 2ResMed To Announce Fourth Quarter And Fiscal Year-End 2014 Results 2BC Technical Partners With MiE America To Be Authorized Provider Of Systems & Service 2
... Sept. 1, 2011 Hill-Rom Holdings, Inc., (NYSE: ... management presentation at the Morgan Stanley Global Healthcare Conference ... 8:35 a.m. ET. You are invited to ... at http://ir.hill-rom.com/events.cfm or access it directly at ...
... makers of the ACCU-CHEK® portfolio of diabetes management products ... Infusion Site Awareness Week, Aug. 29-Sept. 4, a week-long ... management for the 400,000 people in the United States ... "The inaugural Infusion Site Awareness Week, which ...
Cached Medicine Technology:Roche Diabetes Care Celebrates Second Annual Infusion Site Awareness Week, Aug. 29-Sept. 4 2Roche Diabetes Care Celebrates Second Annual Infusion Site Awareness Week, Aug. 29-Sept. 4 3
(Date:7/12/2014)... July 12, 2014 Utah Valley Entrepreneurial ... startups, with awards being bestowed to companies in revenue, ... outstanding entrepreneur community with recognition for new ideas as ... with incoming revenue. , 25 Under 5 highlights outstanding ... that are under 5 years old. Award recipients were ...
(Date:7/12/2014)... Restore My Vision Today is a new ... couple boasting very impressive credentials on the field of ... as a way to help consumers do without the ... respective patients’ treatments . , In particular, ... of pertinent information relevant to eye care like:, ...
(Date:7/11/2014)... 12, 2014 Recently, MagicQuinceaneraDresses.com, the popular ... new collection of 2014 Quinceanera dresses . The ... want to look fashionable without paying a large amount ... up to 57% off. , The company’s online shop ... get amazing special occasion outfits. In addition to exquisite ...
(Date:7/11/2014)... New York, New York (PRWEB) July 11, 2014 ... ( http://www.transvaginalmeshlawsuithelp.com/ ) , filed against Johnson & ... U.S. courts, Bernstein Liebhard LLP notes that the ... is investigating the company’s marketing of surgical mesh ... stress urinary incontinence. According to a report from ...
(Date:7/11/2014)... from Scientology Churches and Missions are taking ... lectures, forums and conferences, information stands, sports events, and ... , Drug abuse is an international epidemic. According to ... alcohol and illicit drugs costs more than $425 billion ... healthcare. , In the face of statistics such ...
Breaking Medicine News(10 mins):Health News:SquareHook is Recognized for UVEF 25 Businesses Under 5 Years-Watch Out 2Health News:Restore My Vision Today | Groundbreaking Book Reveals How To Restore Your Vision Without The Use of Glasses – Vinamy 2Health News:Restore My Vision Today | Groundbreaking Book Reveals How To Restore Your Vision Without The Use of Glasses – Vinamy 3Health News:Cheap Quinceanera Dresses Now Available At MagicQuinceaneraDresses.com 2Health News:Transvaginal Mesh Lawsuit News: Bernstein Liebhard LLP Notes Report of Texas Probe into Ethicon Pelvic Mesh Marketing 2Health News:Transvaginal Mesh Lawsuit News: Bernstein Liebhard LLP Notes Report of Texas Probe into Ethicon Pelvic Mesh Marketing 3Health News:Transvaginal Mesh Lawsuit News: Bernstein Liebhard LLP Notes Report of Texas Probe into Ethicon Pelvic Mesh Marketing 4Health News:Scientologists Taking Action against Drug Abuse 2Health News:Scientologists Taking Action against Drug Abuse 3
... yet another reason to reduce childhood obesity it may ... of the Journal of Allergy and Clinical Immunology ... of having some kind of allergy, especially to a food. ... Health Sciences (NIEHS) and the National Institute of Allergy and ...
... Healthcare Management Systems, Inc.(R) (HMS) announced today it ... technology provider. HMS has grown throughout the years ... currently develop, sell and support integrated clinical and financial ... decade, HMS has expanded its primary market of community ...
... in the May issue of the Journal of the ... electronic prescribing systems may allow for greater efficiency at hospitals, ... of care. , In an attempt to reduce or ... called for the use of electronic prescribing systems in all ...
... PHILADELPHIA, May 4 Children hospitalized with concussions ... clinician in a follow-up exam before returning to ... at The Children,s Hospital of Philadelphia. Rather than ... trouble signs after the initial treatment, the Children,s ...
... May 4 On Monday, May 4, Andrews ... Center at Fort Worth, will receive the "2009 ... Institute, a national women,s health conference for hospitals/health ... planning professionals. The conference offers profiles of successful ...
... Analytical, based in Ames, IA will reveal the latest results ... system. This instrument rapidly identifies microorganisms in less ... The generated fingerprint when compared to a database of ... strain typing and tracking.Because of its ease of use and ...
Cached Medicine News:Health News:New data analysis shows possible link between childhood obesity and allergies 2Health News:Healthcare Management Systems Celebrates 25 Years 2Health News:Electronic prescribing systems boost efficiency, may lead to improved quality of care 2Health News:Children with Concussions Require Follow-up Care Before Returning to Play, Say Researchers 2Health News:Children with Concussions Require Follow-up Care Before Returning to Play, Say Researchers 3Health News:Andrews Women's Hospital Receives National Recognition At Achieving Success In Women's Health Conference 2Health News:Advanced Analytical Reveals New Platform for Rapid Genotypic Identification of Microorganisms at ASM 2009 2
... X-22 Series is up to 10 inches/ 25 cm ... 11 rotors, the Allegra X-22 Series is the space-saving ... Only 18 inches / 46 cm wide • 11 ... 3 different ways to Spin 1.5 mL & ...
... centrifuges offer high-volume versatility along with a ... them ideal for a wide variety of ... Four- or six-place sealed swinging bucket rotor, ... offered , Lare capacity (4 x 750 ...
... performance centrifuge designed to accommodate over 55 ... refrigerated for heat labile samples. Incorporating the ... capability with automatic quick cooling from 22 ... min, no maintenance brushless motor, low pitch ...
Inquire...
Medicine Products: