Navigation Links
BioMarin's Clinical Trial Application for GALNS for Morquio A Syndrome Accepted by the MHRA
Date:3/18/2009

NOVATO, Calif., March 18 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that its application for clinical trial authorization (CTA) 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, has been accepted by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA). BioMarin expects to initiate a Phase 1/2 clinical trial in the next few weeks.

"There are a significant number of untreated Morquio patients in clinics around the world who are very anxious to receive a new treatment option. We are moving forward expeditiously with our GALNS program in the hope of bringing those patients the much-needed treatment they deserve. BioMarin has successfully advanced two enzyme replacement therapies from IND filing to FDA approval in approximately five years each and we plan to leverage our clinical, manufacturing and regulatory expertise to develop this treatment for Morquio A syndrome," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Enrollment in the MorCAP study initiated last November is continuing and we continue to add additional sites. We expect that this survey study will yield valuable information on Morquio patients and will aid in our scientific understanding of the disorder as we further pursue this program."

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. During the dose escalation phase of the study, subjects will receive weekly intravenous infusions of BMN 110 in 3 consecutive 12-week dosing intervals. The objectives of the Phase 1/2 study will be to evaluate safety, pharmacokinetics, pharacodynamics and to
'/>"/>

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

Page: 1 2 3 4

Related biology technology :

1. Dendreon Announces Publication of Phase 1 Study Highlighting Immunologic and Clinical Activity of Lapuleucel-T (Neuvenge(R)) in Advanced Breast Cancer Patients
2. EpiCept Reports Expansion of Clinical Development For Licensed Cancer Compound
3. Romark Initiates Clinical Trial of Alinia(R) for Chronic Hepatitis C in the United States
4. The Multiple Myeloma Research Consortium (MMRC) and Proteolix Initiate Phase 2 Clinical Trial Investigating Carfilzomib for the Treatment of Multiple Myeloma
5. PNH Patients Treated with Soliris(TM) Experienced Dramatic Reduction in Blood Clots During Clinical Trials
6. Clinical Data Acquires Epidauros Biotechnologie AG, Significantly Enhancing Proprietary Genetic Biomarker Portfolio
7. ExonHit Therapeutics: Allergan and ExonHits First Collaboration Compound to Begin Human Clinical Trials
8. Sunesis Pharmaceuticals Announces Reorganization to Focus Resources and Build Value in Clinical-Stage Programs
9. Lorus Advances Clinical Development of GTI-2040 Combined with Ara-C in Acute Myeloid Leukemia
10. YM BioSciences USA cleared by US FDA to initiate Phase II clinical trial of nimotuzumab in children with inoperable, recurrent brain cancer
11. New Study Findings Confirm CLEXANE(R)/LOVENOX(R) Long-Term Clinical Benefit in Patients Suffering From Acute Coronary Syndrome
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:8/1/2014)... analog of topological electron flow in certain semiconductors. ... edge of the material but not through the bulk. ... encounter impurities in the material the electrons will continue ... In the photonic equivalent, light flows not through ... consisting of an array of tiny glass loops fabricated ...
(Date:8/1/2014)... RURO, Inc., Laboratory Information Management ... announces the opening of its first international office. ... District in Shanghai, PRC, RURO Shanghai’s doors are ... of Mr. Shen Gang, the former Technical Director ... years, RURO’s has installed FreezerPro-based solutions in more ...
(Date:8/1/2014)... 2014 According to a new ... - Global Industry Analysis, Size, Share, Growth, Trends and ... USD 25.16 billion in 2013, which is expected to ... CAGR of 10.4% from 2014 to 2020. ... Increasing demand for accurate early diagnosis and ...
(Date:8/1/2014)... 1, 2014 Glencoe Software, Inc. ... "Data InPress" ( http://glencoesoftware.com/data-inpress.html ), a suite ... image data into on-line publications.  The suite ... Injection Service. Photo - ... The DataViewer is ...
Breaking Biology Technology:On-chip topological light 2On-chip topological light 3RURO Opens Office in Shanghai, China 2Global Biophotonics Market is Expected to Reach USD 50.20 Billion in 2020: Transparency Market Research 2Global Biophotonics Market is Expected to Reach USD 50.20 Billion in 2020: Transparency Market Research 3Global Biophotonics Market is Expected to Reach USD 50.20 Billion in 2020: Transparency Market Research 4Glencoe Software Launches Data InPress 2Glencoe Software Launches Data InPress 3
... individuals are invited to enter the 35th ... benefits coming from research and engineering of new ... Products Awards are given in four major categories: ... (500-999 employees); medium companies (50-499 employees); and small ...
... gave about $400,000 worth of software to ... youth and their families. Rob Busch, enterprise sales manager ... at the non-profit's Synergy School in Milwaukee for the ... to upgrade Lad Lakes network and software infrastructure, including ...
... Wis. If Wisconsin is to attract the ... it first has to focus on its own environment, ... Biotechnology and Medical-Devices Association. , ,Part of building the ... large corporations, said WBMA President and EraGen CEO Irene ...
Cached Biology Technology:Panel stresses drawing outside biotech firms to state 2
(Date:8/1/2014)... at The Scripps Research Institute (TSRI) have identified a ... in brown fat cells. , The study, which ... the Proceedings of the National Academy of Sciences ... fat thermogenesis," which is of great interest to medical ... also protect against diabetes. , "This finding offers new ...
(Date:7/31/2014)... In the United States, natural-gas production from shale ... since 2007. Yet scientists still do not fully ... according to a report in the journal ... As gas extraction continues to vastly outpace scientific ... various organizations and institutions, including Princeton University, concluded ...
(Date:7/31/2014)... Fla. --- By tracing nearly 3,000 genes to ... University of Florida scientists have created an extensive ... use large-scale, next-generation DNA sequencing. , Among ... closely related to small moths than to large ... butterflies evolved. The study also found that some ...
Breaking Biology News(10 mins):Scripps Research Institute scientists find new calorie-burning switch in brown fat 2Scripps Research Institute scientists find new calorie-burning switch in brown fat 3'Fracking' in the dark: Biological fallout of shale-gas production still largely unknown 2'Fracking' in the dark: Biological fallout of shale-gas production still largely unknown 3UF study advances 'DNA revolution,' tells butterflies' evolutionary history 2UF study advances 'DNA revolution,' tells butterflies' evolutionary history 3
... Knut Stamnes, Professor and Department Director of Physics ... has been awarded a NASA grant for work ... satellite. His proposal to "use and further develop ... coupled atmosphere-surface systems" is funded as part of ...
... Senior Design team at Stevens Institute of Technology is ... arise during thoracic surgeries. Five undergraduate Biomedical Engineering students ... of existing catheter design and introduces a potentially profitable ... the efforts of the Stevens Innovative Fluid Extraction System ...
... revealing how age-old irrigation techniques may have boosted the ... an estimated 200 million people today. ... kingdom that was located in present-day Sudan, provides details ... disease across populations in ancient times, and how human ...
Cached Biology News:Dr. Knut Stamnes at Stevens receives NASA grant for environmental monitoring 2Dr. Knut Stamnes at Stevens receives NASA grant for environmental monitoring 3Stevens thoracic catheter senior design team takes 1st place at regional ISPE competition 2Stevens thoracic catheter senior design team takes 1st place at regional ISPE competition 3Mummies tell history of a 'modern' plague 2Mummies tell history of a 'modern' plague 3
AVOID FREEZE/THAW CYCLES. Recognizes the ~32 kDa DNase IIα chain and the ~40 kDa proenzyme. DNase II causes both DNA condensation and DNA fragmentation. ...
BD BioCoat Vented Caps for 175 cm2 Flasks...
...
I-Ab MHC Class II...
Biology Products: